Health Care Organization and Use
Patients with sickle cell disease often feel like the ugly stepchildren in the health care setting.
—Tosin Ola (Open Session Panelist)
Chapter 4 described the complications and current management of sickle cell disease (SCD). This chapter discusses the health care system and the use of its services. As with other aspects of SCD, there are information gaps on health care use. Generally speaking, studies have been geared toward economic analyses related to cost rather than detailed descriptions of the services. Thus, it is often unclear what types of services were received, where they were delivered, who delivered them, and what models of delivery were employed.
Children and adults with SCD require both specialty and primary care services continuously across the life span. The unique aspects of care outlined in Chapter 4 often lead to an unpredictable need for urgent and emergency care to manage pain, fever, and neurologic symptoms as well as more predictable needs for care related to screening, early detection, and the management of chronic complications. A broad array of specialty services is required, including multidisciplinary expert providers, medical subspecialists, primary care providers (PCPs), surgeons, anesthesiologists, radiologists, social workers, behavioral health specialists, care coordinators, and community health workers (CHWs). Community-based organizations (CBOs) are essential to this care, as they support patient and community education, provide counseling, and address barriers to care by providing services ranging from transportation to care coordination. Provision of high-quality care that aligns with the six domains of health care quality (addressed in Chapter 6)1 is critical. Furthermore, it is important that health care providers understand and consider the negative societal views of members of the SCD population, including stigma, racism, and various stereotypes, such as the assumption that someone coping with chronic pain is drug seeking (addressed in Chapter 2). Such views not only influence how care is provided but also who provides that care and where. The published literature often omits these subtleties, but the committee believes these aspects should be captured when considering how best to provide high-quality health care to individuals with SCD.
HEALTH CARE FOR CHILDREN WITH SCD
SCD affects children and adults in different ways. This is partly because children’s bodies are progressing through developmental stages, while adult bodies change much less quickly and profoundly, and also simply a matter of time—adults with SCD have accumulated years of damage, which may result in a variety of consequences. Furthermore, children
1 The six domains of health care quality are (1) safety, (2) effectiveness, (3) accessibility/timeliness, (4) person centeredness, (5) efficiency, and (6) equity (IOM, 2001b).
and adults have different capacities for understanding, making decisions about, and managing the disease. Thus, children require different types of care than adults.
Like all children, children with SCD should receive excellent primary care. According to the American Academy of Pediatrics (AAP), pediatric primary care encompasses:
health supervision and anticipatory guidance; monitoring physical and psychosocial growth and development; age-appropriate screening; diagnosis and treatment of acute and chronic disorders; management of serious and life-threatening illness and, when appropriate, referral of more complex conditions; and provision of first contact care as well as coordinated management of health problems requiring multiple professional services. (AAP, 2011)
The content for well-child visits regarding health supervision, anticipatory guidance, monitoring physical and psychosocial development, and appropriate periodic screening and immunizations is delineated in Bright Futures publications.2 The Early Periodic Screening, Diagnosis and Treatment (EPSDT) benefit also establishes requirements for children under age 21 on Medicaid to receive screening and treatment services. Although the two approaches certainly overlap, EPSDT emphasizes hearing, vision, and dental screening and treatment. In addition to routine well-child care, children with SCD must also receive specialized preventive care, as discussed in Chapter 4.
Providing the broad array of services needed by children with SCD requires a different model than that of routine pediatric health care. For children who are unable to access specialized sickle cell care centers, the medical home (discussed later in this chapter), a model formulated by AAP, is one model of care available for children with SCD. Ideally, a medical home provides care that is accessible, continuous, comprehensive, family-centered, coordinated, compassionate, and culturally effective to every child and adolescent. A pediatric medical home is a family-centered partnership within a community-based system that provides uninterrupted care with appropriate payment to support and sustain optimal health outcomes. Medical homes address preventative, acute, and chronic care from birth through transition to adulthood. “A medical home facilitates an integrated
2 Bright Futures publications are geared toward health professionals and educators and cover topics such as health supervision, nutrition, and mental health for infants and children (Bright Futures, n.d.).
health system with an interdisciplinary team of patients and families, PCPs, specialists and subspecialists, hospitals and health care facilities, public health and the community” (AAP, n.d.).
In general, the literature supports the utility of the medical home model in improving health outcomes, reducing unneeded use of care, and improving family communication and functioning for children with chronic illness (Kuhlthau et al., 2011). Having a PCP, however, is not equivalent to a medical home for children with SCD, as the PCP may not offer many of the components needed (Liem et al., 2014; Raphael et al., 2013b) or provide the specific preventive services for optimal outcomes (Bundy et al., 2016). In these situations, it may be necessary for providers to create a “medical neighborhood” in which hematologists and PCPs closely collaborate to provide children with necessary care (Raphael and Oyeku, 2013).
Establishing the connections among providers necessary to assure more comprehensive care can be difficult due to the long wait times for available appointments (Martin et al., 2018) or inadequate referral patterns (Bundy et al., 2012; Martin et al., 2018). In a sample of children recruited in a sickle cell center or hospitalized for SCD, Raphael et al. (2013a) reported that children receiving comprehensive care from a medical home, as measured by specific, standardized questions eliciting parental report, had fewer emergency department (ED) visits and hospitalizations (Raphael et al., 2013a). However, the source of care was not further characterized. Rattler et al. (2016) conducted a study to examine the coordination of care and found that most caregivers reported having a PCP for their child, whereas only 25 percent of caregivers reported having access to coordinated care. The study also found that coordination of care resulted in 88.1 percent of caregivers feeling satisfied about the communication occurring between medical providers; however, only 67.3 percent of caregivers reported being satisfied with the communication occurring between doctors and nonmedical service providers.
Other studies report that a comprehensive approach to care, especially an approach involving hematology, results in greater receipt of special preventive services for children with SCD (Bundy et al., 2016; Martin et al., 2018). Part of the association between more comprehensive care and reduced acute care use may reflect greater adherence to medication prescriptions among those with preventive visits (Walsh et al., 2014).
Another important factor in assessing the effectiveness of the medical home is the variation in the components that are implemented. In an evaluation of the medical homes for a sample of children with SCD, care coordination was more likely to be experienced than accessibility or comprehensiveness; however, the same study found that accessibility and coordination were associated with fewer ED visits (Liem et al., 2014).
Children with SCD require a wide array of services for health maintenance, including (1) therapeutic interventions to prevent complications overall and in high-risk subpopulations, (2) screening for high-risk features and early detection of chronic complications, and (3) long-term disease-modifying therapies to decrease complications. Preventing infection, stroke, and acute pain and acute chest syndrome (ACS) episodes by starting hydroxyurea therapy early in life for children with the SS and Sb0 thalassemia types has been the mainstay of care. In addition, early treatment for acute febrile illness and acute pain has been the focus of acute complications. Detecting bacteremia and sepsis early leads to better outcomes by preventing morbidity and mortality. Aggressive treatment of acute pain and alleviating pain early may also decrease complications, such as ACS. Early assessment of pain and fever may detect other acute complications, such as ACS, splenic sequestration, and exacerbation of anemia. These services are provided in many settings but have traditionally developed in large, urban, academic hospitals. Emergency medicine teams are actively involved in managing these pediatric patients, but acute care by hematologists and other sickle cell experts has emerged as well. Infusion centers (discussed below) and acute care units staffed by experts allow for the rapid assessment and treatment of SCD complications without involving or with minimal support from emergency medicine.
The use of ambulatory subspecialty services has not been reported. In addition, the use of preventive care services unique to SCD, such as transcranial Doppler (TCD) screening, cannot always serve as a surrogate marker of subspecialty service use because children may be receiving care from a sickle cell expert but either the screening has not been ordered or appointments have not been kept. As an example, implementing TCD ultrasonography services to detect SCD-related stroke risk, specifically in children ages 2–16 years, started shortly after results of the multicenter trial recommended chronic transfusions for children at high risk for stroke based on TCD measurement of time-averaged mean of the maximum velocity in middle cerebral arterial circulation (Armstrong-Wells et al., 2009). However, recent data from six state Medicaid programs suggest that as few as 44 percent of children who require stroke risk screening actually receive it (Reeves et al., 2016).
As noted in Chapter 4, children with SCD experience repeated episodes of acute illness, such as pain, fevers, and aplastic crises, requiring ED visits or hospital admission. According to national estimates, children with SCD
average about 0.6 ED admissions and at least one hospital admission per year (Brousseau et al., 2010). Approximately one-third of children with SCD will have neither an ED visit nor a hospital admission in any given year, whereas slightly more than 10 percent will have three or more such episodes. Data from a retrospective cross-sectional descriptive analysis of administrative medical claims found that in Texas, 37–43 percent of children with SCD have at least one ER visit per year, a figure that is consistent with the previous statement (Raphael et al., 2009).
A significant issue concerning acute care is that individuals with SCD often have repeat ED visits and admissions. For example, one study found that among children aged 1–9 years, 4.1 percent (95% confidence interval [CI] 3.6–4.6) of hospitalizations were followed by a return for a treat-and-release ED visit within 30 days after hospital admission and that 12.8 percent (95% CI 12.0–13.6) of hospitalized children had a second hospital admission within 30 days of the first admission. For all children with SCD between the ages of 10 and 17 years old, the corresponding rates were 6.9 percent (95% CI 6.4–7.4) and 23.4 percent (95% CI 22.5–24.3) (Brousseau et al., 2010). From 2009 to 2014, the most common diagnosis among children associated with a subsequent, unplanned readmission to the hospital was SCD, and SCD had the highest percentage of hospital readmissions overall. Thus, this study found that SCD was among the 10 most resource-intensive diagnoses for admission within the study population (Heslin et al., 2018). However, these results are from one study only, so care must be taken not to overstate these findings.
As noted in Chapter 4, oral infections and gum disease are triggers for chronic inflammation and pain and are underlying factors for health care visits. Those with SCD may be more prone to dental problems due to hypoxia in the dentin following sickle crises, and individuals with SCD present with higher rates of dental caries than individuals in the general population (Laurence et al., 2006). Individuals with SCD may also present with delayed tooth eruption and pulpal necrosis that increase with age (Costa et al., 2013, 2016). In a population of 250 children and adolescents with SCD, 47 percent had caries and 14 percent had periodontal problems (Luna et al., 2018).
A retrospective analysis of 10 years (2000–2011) of dental records for 574 individuals with sickle cell anemia at the Center of Hematology and
3 Because there are few data about dental care for individuals living with SCD at any age, research discussed in this section involves adolescents and adults.
Hemotherapy in Maranhao in Brazil was conducted to describe the use of dental services over time. The study population consisted primarily of children and young adults and a small number of adults. The study findings showed that teeth filling and extractions, periodontal treatment, and endodontic treatments were prevalent in the young adult and adult populations, signaling an increase in the need for these procedures as individuals with SCD age. The study authors speculate that the procedures could have been avoided with a care plan based on prevention and health promotion (Costa et al., 2016).
Importantly, the U.S. public insurance system does not universally provide dental coverage, even in Medicaid plans, for children or adults (Berdahl et al., 2016; Naavaal et al., 2017). Thus, there may be significant barriers to dental care access for patients with SCD. In one analysis of national data, Laurence et al. (2013) found that among patients having a sickle cell crisis, those with dental infections were 72 percent more likely to be admitted than were those not having dental infections (prevalence ratio [PR] = 1.72, 95% CI 1.58–1.87). One pilot study found that adults with SCD receiving free dental care demonstrated a significant reduction in the number of hospital admissions as well as in the total number of days spent in the hospital if dental work was completed; however, the study also found that there was an increase in the number of hospital days experienced by men in the study population (Whiteman et al., 2016). A recent Cochrane review did not identify any randomized controlled studies that evaluated interventions to treat dental complications in SCD, indicating a significant research gap and the need for randomized controlled studies in this area (Mulimani et al., 2016).
As discussed in Chapter 2, children and youth with SCD experience difficulties in school and may have poorer academic performance, as indicated by grade retention or lower academic grades. This increased risk for learning difficulties may make them eligible for special educational assistance.
Special Education System
Services for children with special educational needs are made available under the Individuals with Disabilities Education Act (IDEA), most recently reauthorized under the Every Student Succeeds Act in 2015. IDEA is meant to ensure that all children with disabilities have access to a free appropriate public education that emphasizes special education and related services designed to meet their unique needs and prepare them for further education, employment, and independent living (ED, n.d.). Services for children
ages 0–2 are provided under Part C of the act (often referred to as early intervention), and older children are covered under Part B. Services provided through the educational system end at 21 years old. Thereafter, youth and young adults with significant disabilities may be eligible for vocational and rehabilitation services if these may improve their ability to obtain employment (Linebaugh, n.d.). In higher education, Section 504 and Title II of the Americans with Disabilities Act prohibit discrimination due to disabilities and require reasonable accommodations (ED, 2020). However, services available to individuals who are more than 21 years old are not as plentiful as those offered to individuals with SCD while they are in the primary and secondary education school system.
Early Intervention (Part C of IDEA)
This is a state-run program with funding from the federal government. To be eligible, children
- should be experiencing developmental delays, as measured by appropriate diagnostic instruments and procedures, in one or more of the following five areas: cognitive development, physical development, communication development, social or emotional development, or adaptive development; or
- have a diagnosed physical or mental condition that has a high probability of resulting in developmental delay (ED, 2016).
Services under Part C must meet certain minimal criteria and can be delivered through a variety of state agencies. However, the minimal criteria refer primarily to the processes that the state must implement and do not specify quality. States also have leeway to specify what conditions qualify for early intervention, especially under the “at-risk” rubric.
Children with SCD could clearly qualify for early intervention under “at-risk” criteria. SCD falls under the general category of genetic conditions but is not specified as a qualifying condition (Office of Special Education Programs, 2011). Access to early intervention would require a developmental assessment by the PCP or other provider and referral to early intervention services if developmental problems were noted. This strategy requires a problem, is not necessarily preventive, and places an additional burden of proof on the family, further reducing access to services. The committee found no literature examining early intervention for children with SCD. In general, for special needs populations, such access depends on the restrictiveness of the state criteria and the severity of dysfunction (McManus et al., 2009, 2019). Having a medical home does not improve referrals to Part C of IDEA (Ross et al., 2018).
Special Education Schools (Part B)
As with early intervention, to be eligible for a special education school, a child must have a disability that falls in 1 of 13 categories and need special services to succeed in school. Federal funding is provided to states to pay for the estimated increased cost of special education. The funding is primarily distributed to public schools except for when a private school provides the required services, thus making it eligible for funding. An additional requirement is that the child have a free and appropriate public education.
During their primary and secondary educational careers, children and youth with SCD are more likely to exhibit lower academic attainment, performances (as measured by receipt of special education services), and retention rates than the reported national averages, as well as lower attainment, performances, and retention rates than those reported for healthy African American children (Crosby et al., 2015; Epping et al., 2013; Fowler et al., 1988). Some of these trends are a reflection of the direct impact of SCD on academic attainment, performance, and retention due to frequent hospitalizations, SCD-related neurocognitive complications, and medical visits. Socioeconomic adversity also plays a role, as do the characteristics of the educational support available to the child while at home (Ladd et al., 2014). As with early intervention, however, a child receiving special education services does not mean that those services are adequate to the child’s needs. One study found that a multidisciplinary intervention to improve school performance in children with SCD succeeded in increasing the proportion with an individualized education program, a requirement of special education, but did not affect grade retention or absenteeism 2 years after the intervention (King et al., 2006). The major driver of the results was the child’s intelligence quotient, suggesting that improving academic performance may require preventing brain injury.
Evidence from Children with Special Health Care Needs and with Medical Complexity
Because of the sparsity of literature directly related to SCD, the committee reviewed evidence from the general literature on children with chronic or complex conditions to inform their understanding of the medical needs of children with serious chronic conditions. Children with SCD fall under the rubric of children with special health care needs (CSHCN), who are defined as “having or being at increased risk for chronic physical, developmental, behavioral, or emotional conditions and who also require health and related services of a type or amount beyond that required by children generally” (McPherson et al., 1998, p. 138). Systems of care for
CSHCN are dependent on ensuring that families are partners in care; that there is early and continuous screening; access to a medical home to provide community-based, coordinated care; adequate insurance and funding to cover services; and a plan for families and providers to help patients transition to adult care and services (HRSA, 2019). For children and youth with SCD, such systems would include the routine primary care necessary for all children but also access to the special preventive services needed for SCD, emergency and hospital services for acute complications, and the specialists and rehabilitative services required to improve functioning.
Children with SCD may also qualify as children with medical complexity (CMC). Within the pediatric population this group is important because of the high costs of care, unmet health needs, variability in the quality of care received, and potentially poorer health outcomes, all of which are associated with their diagnosis. Children with SCD can be identified at a population level through the use of administrative data, and survey data may help characterize any confounding that may occur as the result of the presence of one or more chronic conditions (Berry et al., 2015).
The impetus behind CMC and CSHCN classification schemes is that chronic conditions in children tend to be relatively rare illnesses. While the management of the individual conditions may be quite specific, chronic conditions share a number of common characteristics. For example, they may require coordinating several types of services and specialty providers, place substantial burden on caregivers to organize and actually provide care in the home, incur increased cost depending on insurance and other sources of support, and require a specific plan to transition to adult care. Information is lacking on some of these issues for SCD, so the committee relied on the more general literature to illustrate concerns.
Data about the use of services by CSHCN and CMC can be obtained from national surveys. The National Survey of Children’s Health (Data Resource Center for Child and Adolescent Health, n.d.) and its predecessors use a specific algorithm to identify CSHCN, whereas other national datasets may rely on parental report of “fair or poor health.” In addition to national surveys, data on health care use may also be obtained from other administrative or medical records databases such as hospital discharge records (Grosse et al., 2010).
The research on CSHCN supports the importance of a medical home arrangement in improving physical and mental health outcomes, satisfaction with care, accessibility, efficiency in health services use, access, systems, and communication. The effects of a medical home arrangement on family functioning and family costs were less consistent (Kuhlthau et al., 2011). When coordinated care is provided through state insurance plans (e.g., Medicaid and State Children’s Health Insurance Program managed care),
the results are mixed (Huffman et al., 2010). One study found that those with managed care plans experienced fewer unmet needs (including dental care) than those without managed care plans, but health care use was similar between the two groups. However, the study also found that plans that provided specialty services through carve-outs had decreased access to such services. Parental satisfaction was also less because of the limitations in the providers who were in the plans (Huffman et al., 2010).
Despite the potential advantages of a medical home, there are variations in access to this type of coordinated care. While access to coordinated care is similar between those with and without special needs (about 43 percent versus 53 percent, respectively), only 23 percent to 36 percent of children had a medical home if they were from a home where English was not the primary language or where parents exhibited low educational attainment or had low income (Lichstein et al., 2018). CSHCN with private insurance were more likely to have a medical home than CSHCN with any public insurance (about 52 percent versus 36 percent, respectively) (Lichstein et al., 2018).
In terms of access to health services, the literature generally supports an independent association between minority status and Medicaid insurance, with minority children having lower rates of established PCPs and preventive well-child or dental care visits (Berdahl et al., 2016; Elixhauser et al., 2002). While children who are reported to be in fair or poor health have higher outpatient use and more prescriptions, it is unclear whether this is in proportion to their medical needs (Elixhauser et al., 2002). Minority children are also less likely to receive early educational interventions, regardless of the strictness of the eligibility criteria in the state. The literature also provides evidence that the earlier and more intense the early intervention, the better the outcomes (Litt et al., 2018; McManus et al., 2019); unfortunately, some studies found that children from minority groups or children whose mothers did not speak English received less intensive early intervention services (McManus et al., 2019; Richardson et al., 2019).
Access to subspecialty services is dependent on the availability of subspecialists in the community, which varies substantially across the United States. Subspecialty service availability is one area in which African American children have a slight advantage over non-African American children, as the concentration of African American children in urban areas aligns with the high concentration of specialty services in corresponding areas (Ray et al., 2014). Ray et al. (2014) found that children in areas of low to moderate concentrations of specialists report more mental health problems. Unmet needs in the low specialty areas often reflected a lack of providers and the need for transportation (Ray et al., 2014).
Enabling services are defined as
non-clinical services that do not include direct patient services that enable individuals to access health care and improve health outcomes. Enabling services include case management, referrals, translation/interpretation, transportation, eligibility assistance, health education, environmental health risk reduction, health literacy, and outreach. (HRSA, n.d., p. 2)
Case Management and Care Coordination
Among the most frequently cited enabling services are studies and descriptions of care coordination/case management. Pordes et al. (2018) provide a useful framework for coordination in medical complexity. They postulate three basic care models: the primary care-centered (PCC), consultative- or co-management-centered (CC), and episode-based (EB) models. PCC is analogous to the medical home. CC is the familiar reliance on specialty clinics to coordinate care with an individual’s PCP; this model acts as a bridge between primary and tertiary care. EB is dependent on coordination around a specific episode of care (e.g., hospitalization) to facilitate transitioning the child to the home. Case management services may improve acute pain and chronic pain management, but their use is under-reported (Brennan-Cook et al., 2018).
The CC model would appear more appropriate for SCD, as Pordes et al. (2018) note that the target population for this model would include those with rare disorders, novel treatments, or high dependence on medical technology, especially those who may live at a distance from the tertiary center. In one study of medically fragile children, such a model reduced hospitalizations and hospital days and resulted in decreased tertiary center costs (Gordon et al., 2007).
As noted above, the PCC model or medical home for children with SCD has produced mixed results. In more general CMC populations, the PCC model is associated with fewer unmet care needs (Boudreau et al., 2014), less functional disability (Litt and McCormick, 2015), and lower out-of-pocket costs (Porterfield and DeRigne, 2011). Part of the difficulty in obtaining replicable results for researchers examining outcomes associated with the PCC model may be a reflection of the methodological gaps in this research (Pordes et al., 2018) and the complexity of implementing an effective model that may require a number of stakeholders and substantial investment (Berry et al., 2017).
4 It is important to note that adults as well as children are eligible for enabling services.
While it was not their focus, Pordes et al. (2018) acknowledge that care coordination may involve stand-alone case management services provided by an insurance company or community agency. As noted above, the evidence is mixed as to the effect of the medical home on parents’ experience of coordination and reduced use of acute care. In at least one study, the stand-alone model proved less effective (Wood et al., 2009). The committee found no studies comparing different types of case management in the care of children with SCD.
Community Health Workers and Community Educator Counselors
Services provided by CHWs include support for medication adherence, support through transition from pediatric- to adult-focused care services, and care coordination (Green et al., 2017; Wood et al., 2009). The Patient Navigator to Reduce Readmissions study is a single-site, multidiagnoses pragmatic clinical effectiveness trial that compares a multifaceted, stakeholder-supported navigator intervention with usual care processes for hospital-to-home transitions for individuals hospitalized with various conditions, including SCD (Prieto-Centurion et al., 2019). The study uses trained CHWs to conduct in-person visits in the hospital and after discharge, in addition to telephone-based coaching, and compared the results with those from usual care in order to improve the experience of hospital-to-home transition of care and reduce the 30-day readmission rate. While it is still ongoing, this study includes early and continuous patient and caregiver engagement with clinicians and health system administrators in an iterative process to determine the behavioral components that are critical to success (Prieto-Centurion et al., 2019). Additional information on the role that CHWs play in SCD care can be found in Chapter 8.
A lack of transportation services is a significant barrier to health care use in patients with chronic health conditions (Syed et al., 2013). The use of transportation services is reported to state funding authorities for individuals living with SCD; however, this information is not published. Access to information about the need for transportation services and the type of services used would be useful in improving our understanding of barriers to care and developing funding sources. The need for transportation to and from medical appointments and following hospitalizations is expected, but further investigation is warranted into whether transportation to pharmacies, dental visits, and behavioral health appointments could benefit this population.
Telehealth comprises the broader consumer-facing methods or means of support provided to patients to enhance health care delivery and clinical outcomes (Ray and Kahn, 2020). Telemedicine, a type of telehealth, is the use of technology to provide clinical care from a provider to a patient. Telehealth support helps improve access to care by filling a gap that traditional models of health care delivery have exposed (Ray and Kahn, 2020).
Telehealth can also prove vital in addressing the psychosocial needs of people living with SCD. An interactive mobile monitoring system using text messaging via a web-based platform to deliver acceptance and commitment therapy,5 a form of cognitive behavioral therapy (CBT), has been shown to provide real-time psychotherapy intervention to adolescents living with SCD (Cheng et al., 2013). The final results of this study have not been released, but a similar intervention showed significant favorable effects for depression, mindfulness, and other psychological symptoms in individuals with chronic pain (Yang et al., 2017), suggesting the need for more definitive studies in SCD.
Two recently funded clinical trials leveraging telehealth among transition-age individuals with SCD are currently ongoing. One study compares the effectiveness of two self-management support interventions (by CHWs and the mobile health iManage app) versus enhanced usual care6 (Children’s Hospital of Philadelphia et al., 2018). The second study compares the effectiveness of a structured, education-based transition model with or without virtual peer mentoring through a web-based platform (Osunkwo and PCORI, 2018). Both studies will evaluate the role of the interventions in improving health-related quality of life (HRQOL) and acute care use.
A recent meta-analysis of the literature on telehealth in SCD management over the past two decades describes interventions for 747 participants, including older children and adolescents (69 percent) and adults older than 18 years of age (31 percent), via text messaging (25 percent), native mobile (19 percent), or web-based apps (31 percent) in addition to mobile direct observation (13 percent) and Internet-delivered CBT therapy (13 percent), interactive gamification (13 percent), and electronic pill bottles (6 percent) (Badawy et al., 2018a). Telehealth interventions targeted mostly medication adherence (31 percent); self-management, pain reporting, and symptom reporting (44 percent); stress, coping, sleep, and daily
5 The focus is on enabling patients to accept troublesome thoughts instead of fighting to reduce them (Guarna, 2009).
6 Usual care is a condition in which health care personnel determine a patient’s care independent of a research team; enhanced usual care is when usual care is improved (enhanced) by using research protocols (Freedland et al., 2011).
activities reporting (25 percent); cognitive training for memory (6 percent); SCD and reproductive health knowledge (31 percent); CBT (13 percent); and guided relaxation interventions (6 percent) (Badawy et al., 2018a). While various telehealth modalities are being used in SCD management with demonstrated feasibility and acceptability, outcomes data on efficacy are modest, as study sample sizes have been small (Anderson et al., 2018; Cady et al., 2009). Future research should particularly be directed toward enhancing disease education, health literacy, and facilitating positive health behavior changes (Issom et al., 2015).
The concept of “telementoring” refers to virtual, case-based peer learning in a hub-and-spoke model by bringing together teams of disease experts and other health care professionals who manage the individuals locally, increasing provider knowledge and comfort, and equipping providers with disease-specific expertise. The telementoring model has been applied through the Health Resources and Services Administration (HRSA), which funded the Sickle Cell Disease Treatment Demonstration Regional Collaborative Program (SCDTDRCP) to expand access to care by providing the local care team with access to expert consultation and peer dialogue around disease management questions (Shook et al., 2016; Stewart et al., 2016). While it is too early to evaluate its impact on health outcomes for SCD, the educational impact of telementoring models has been demonstrated to be feasible and effective in educating providers on delivering specialty care from a distance and improving health care delivery (Salgia et al., 2014).
While the committee found no publications addressing access to language translation services for SCD management, the population of interest consists of racial and ethnic groups whose first language is not necessarily English. There are increasing numbers of Spanish-speaking individuals living with SCD in the United States. Most states also have a high proportion of African and European immigrants with SCD who do not speak English as their first language. Being in a home where English is not the primary language does not necessarily result in language delay; the child may be up to date in the other languages. However, children may be slower to speak if toggling between English and another language, but eventually they become fluent in both. Furthermore, one study found that parents with limited English proficiency were more likely to report that their CSHCN were uninsured and had no usual source of care or medical home (Eneriz-Wiemer et al., 2014).
It is well known that children with language and cognitive delays have early-onset school difficulties (Cheng et al., 2014) and that these complications are common in children with SCD (Crosby et al., 2015). Therefore, it
is important that there is an assessment for language proficiency in the family of those with SCD, as a failure to recognize this as a barrier may lead to suboptimal care. Health care systems, providers, and public health departments continue to be cognizant of and responsive to the growing diversity of the patients in their catchment area by providing educational materials in multiple languages to meet patients’ needs. For example, the Massachusetts Department of Public Health’s website provides information in at least 12 languages (Commonwealth of Massachusetts, 2020). Providers serving the SCD population can leverage existing translated health care materials prepared for the general patient population. Finally, hearing loss is a known complication of SCD and may be the result of ischemic insults to the cochlea or as a consequence of common SCD medications, transfusional iron overload, and infections (Stuart and Smith, 2019). Alternative forms of language support, such as sign language, may also be needed in this population.
Health Literacy Services
Understanding English does not assure that materials for and communications with those with SCD will be equally well understood and acted on. Educational information provided to persons living with SCD should reflect Centers for Disease Control and Prevention (CDC) recommendations for actionability and understandability as measured by the Clear Communication Index7 to ensure that the information is accessible to individuals with low health literacy (McClure et al., 2016). Clinical experience demonstrates low health literacy among caregivers of children with SCD and adolescents and adults living with SCD (Perry et al., 2017; Yee et al., 2019). Someone with low health literacy can be presumed to have low adherence to clinical care recommendations and low understanding of the disease. However, the committee found few studies that have explored the relationship between health literacy and health care use in people living with SCD. One study suggests that there is no relationship between health literacy and acute care service use (Caldwell, 2019). Large studies with representative samples of individuals living with SCD are needed.
Home Health Care Services
Home health care has been available for children living with chronic conditions since the 1990s. It is rarely covered by private insurance, so enrollment in Medicaid is required. As with many aspects of Medicaid,
7 The Clear Communication Index is a research-based tool developed by the Centers for Disease Control and Prevention to assess public communication materials (CDC, 2019a).
substantial variation exists among states regarding the implementation of the necessary waivers and other mechanisms. Moreover, the low levels of payment for services and the scarcity of skilled workers further limits access to such services. Suggestions for changes include integrating home health services into a child-focused health care system and greater reliance on telehealth support (Foster et al., 2019). The committee found no literature regarding home care services for children with SCD.
TRANSITION FROM PEDIATRIC TO ADULT CARE
More than 95 percent of children with SCD will survive into adulthood due to successes associated with early diagnosis, innovative preventative therapies, and improved comprehensive care (Hassell, 2010; Lanzkron et al., 2013; NHLBI, 2014; Quinn et al., 2010). However, transition to adulthood with SCD is often associated with a loss in the gains made during the childhood period. Changes in the course and consequences of the disease shift from adolescence to adulthood. This is most evident in adult mortality rates and in the patterns of health care usage, particularly in the use of acute care. A major issue for health care providers is to maintain therapeutic continuity across this transition. However, there is limited research to support evidence-based interventions that effectively reduce the high mortality and morbidity associated with the transition period (Hamideh and Alvarez, 2013).
Impact of Transition
Mortality rates among children with SCD have declined significantly. A study by Hamideh and Alvarez (2013), using data from U.S. death certificates from the periods 1999–2009 and 1979–1998, found that mortality rates were significantly decreased in the 1999–2009 period. Compared with the 1979–1998 period, mortality rates had decreased by 61 percent in infants less than 1 year of age, 67 percent in children aged 1–4 years, and 22 to 35 percent in children aged 5–19 years. However, mortality rates for individuals more than 19 years of age were seen to increase from 0.6 in the 15- to 19-year group to 1.4/100,000 in the 20- to 24-year group (Hamideh and Alvarez, 2013). This period corresponds to the transition period from pediatric to adult medical care. Paulukonis et al. (2016) also found increases in mortality rates during the transition period. Based on surveillance data from California and Georgia, they reported a tripling of all-cause mortality among 15- to 24-year-olds compared with those under age 14.
Health Care Use
Where young adults with SCD receive care is another noticeable change during the transition period. The ED becomes a primary site of care for young adults with SCD. An examination of data from 4,636 patients with SCD from the California Registry and Surveillance System for Hemoglobinopathies project found a higher use of acute care services among young adults with SCD. From 2005 to 2014 the average number of annual ED visits for patients with SCD was 2.1; ED use was highest among young adults (2.8 visits for individuals aged 20–29.9 years) (Paulukonis et al., 2017).
Hospitalizations and readmissions are also higher in young adults. Data from the Healthcare Cost and Utilization Project 2000–2016 Nationwide Inpatient Sample showed that hospitalizations and readmissions are also higher in young adults. Adults ages 18–34 with SCD had the highest number of hospital inpatient stays (67,900 stays in 2016) compared with individuals with SCD in any other age groups (less than 26,000 stays for each of the other age groups in 2016) (Fingar et al., 2019). Furthermore, in 2016 the all-cause 30-day readmission rates following initial inpatient stays among patients with SCD were highest among those ages 18–44 (39.4 percent) and lowest among those younger than 18 (20.1 percent) (Fingar et al., 2019).
Continuity of Care
The disparities in health outcomes during transition for young adults with SCD result from a complex interplay of factors related to the patients, their families and social networks, communities, health care providers and health care systems, practice settings, and government policies. The main challenge with transition for any chronic condition of childhood is the loss of continuity in medical care and psychosocial support, which can have a negative impact on short- and long-term outcomes. The committee was unable to find accepted standards for how best to transition young adults with SCD to adult care. However, there have been requests from the SCD community and stakeholders (including providers, health care systems, insurance payers, CBOs, patients, and family members) for a standardized process. There are models and indicators of successful transition, discussed further in this chapter and in Chapter 6, for the general population that could form the basis for an approach for the SCD population.
Barriers to Transition
Several patient, provider, and health system factors contribute to the poor health outcomes of young adults with SCD during transition (Bemrich-Stolz et al., 2015; Sobota et al., 2015; Treadwell et al., 2016). Some young
adults with SCD struggle emotionally with adjusting from a more paternalistic pediatric model to the adult “individualistic” model of care. Adherence to disease-modifying treatment drops significantly as they cope with the developmental maturation process (Blum et al., 1993). Some young adults with SCD report having inadequate information about adult care and a poor understanding of their disease and how it becomes more complex with age (Sobota et al., 2015). Knowledge gaps and poor communication also exist among pediatric and adult providers, compounding issues created by a lack of a standardized patient education curriculum for young adults with SCD (Sobota et al., 2015). Changes or lapses in insurance lead to gaps in comprehensive care—specialized and primary—during transition, amplifying the existing barriers to accessing crucial services as the disease burden is increasing (Crowley et al., 2011).
Models of Transition
In 2011 AAP, in partnership with the American Academy of Family Physicians and the American College of Physicians, co-authored an expert opinion and consensus statement that provided clear guidance and a supportive decision algorithm to describe practice-based recommendations and six core elements for the health care transition of adolescents into adulthood, to maximize their lifelong functioning (AAP et al., 2011). These six elements include transition policy, transition tracking, transition readiness, transition planning, transition and transfer of care, and transition completion (AAP et al., 2011; White and Cooley, 2018). In 2018 the two authors updated their guidance to provide more practice-based quality improvement specificity for the six core elements (White and Cooley, 2018). Further expansion of the six core elements of optimal transition was refined by the Got Transition™/Center for Health Care Transition Improvement, which resulted in an open access comprehensive toolkit for clinicians (ACP, 2019). A time-series comparative study at five large pediatric and adult academic primary care practices in the District of Columbia found that quality improvement activities based on the Got Transition core elements resulted in improvements in transition from pediatric to adult care, as measured with the Health Care Transition Index (pediatric and adult versions) (McManus et al., 2015a). Another pediatric-to-adult managed care transition pilot project for young adults with chronic mental health challenges used the quality improvement process to incorporate the Six Core Elements of Health Care Transition (2.0) into routine care, showing significant improvement in the transition index and all six elements over an 18-month period (McManus et al., 2015b).
Young adults with SCD transitioning to adult care have specific needs. The critical components of transition readiness for patients include an
increase in disease knowledge, independence with self-care skills, and improved pediatric and adult provider transition support (Monaghan et al., 2013). Structured patient education increases disease knowledge, provides self-management skills, and is effective at increasing autonomy, self-efficacy, and disease self-management, which reduces acute complications in juvenile diabetes (Monaghan et al., 2013).
While most transition coordination models use education to address specific patient-level barriers to care (e.g., medication adherence, disease knowledge, transition readiness), it is also important to address the more complex societal ecosystems (e.g., family, school, culture, laws) within which the young adults with SCD must navigate independently in adulthood (Griffin et al., 2013). Health system–based interventions alone are inadequate to address all their needs during transition. Researchers recommend a holistic transition framework that is multifaceted and incorporates the medical, psychosocial, education, vocational, and other needs of young adults as well as the involvement of the primary care and specialty care teams (DeBaun and Telfair, 2012; Treadwell et al., 2011). The impact of CBOs and urgent care teams needs to be explored. Best practices, particularly around transition readiness, optimizing transition success across various care delivery sites, and models of care require further investigation.
Young adults with SCD have also identified isolation as a key challenge during the transition process, and learning from someone who has been through the process is a desirable component of transition (Sobota et al., 2015). The importance of peer mentoring, where an older peer with experience provides support and guidance, was repeatedly mentioned as a desired approach by patient panelists who participated in the National Academies of Sciences, Engineering, and Medicine’s SCD committee’s open sessions. A pilot project on peer mentoring for 40 mentees with SCD demonstrated that peer mentoring can effectively sustain support for young adults with SCD by reducing isolation and improving community engagement and self-efficacy while modeling independent life skills without the high cost of professional support systems (Okochi et al., 2019).
A comparative effectiveness study sponsored by the Patient-Centered Outcomes Research Initiative is currently under way at 14 clinical sites across the eastern United States to examine the effect of a structured education-based transition program with or without peer mentoring (PCORI, 2020). The program was modeled after the six core elements in Got Transition and tailored specifically for pediatric and adult SCD clinics (PCORI, 2020). Sites received coaching using the model for improvement methodology and participated in monthly quality improvement coaching calls. All sites have systematically improved their transition processes, indicated by their scores on an SCD transition process measurement tool that measures
adherence to the six core elements. This model has also proven feasible within large health systems (Jones et al., 2019) and a Medicaid managed care plan (McManus et al., 2015b). The 2016 National Survey of Children’s Health of 20,708 adolescents, aged 12–17 years, found that only 17 percent of CSHCN met the overall transition measure that was calculated based on three elements: whether a health care provider discussed the fact that health care will shift eventually to an adult health care provider, whether a health care provider actively worked with youth to gain self-care skills or to understand that health care will change at age 18, and whether the youth had time alone with a health care provider during the last preventive visit (Lebrun-Harris et al., 2018). Efforts need to be intensified to ensure that all CSHCN, including those with SCD, receive adequate transition planning support (Lebrun-Harris et al., 2018).
In an effort to develop a transition model that works for young adults with SCD, researchers stress that the process must begin early, perhaps even as early as birth, and incorporate the whole life perspective. Areas of emphasis for transitioning should include “preparing pediatric patients for the culture of adult medicine, promoting self-advocacy in obtaining support from schools and employers, and addressing issues of funding of health care services” (Treadwell et al., 2011, p. 119).
HEALTH CARE FOR ADULTS WITH SCD
Just a few decades ago SCD was characterized as a childhood disease because relatively few individuals with the disease lived far into their adult years. However, advances in treatment have led to SCD being characterized as a lifelong, chronic condition. Subsequently, adults with SCD in the United States and other countries require health care that manages and responds to disease-related symptoms. One major factor that distinguishes adults with SCD from children is that adults’ bodies bear the history of years of SCD’s effects. One study found that by the time individuals with SCD reached adulthood, the majority (59.3 percent) had accumulated end-organ damage involving at least one organ and 24.0 percent had multiple organs involved. The number of end organs affected is positively correlated with mortality (Chaturvedi et al., 2018). This makes adults’ health care requirements different in various ways.
Individuals with SCD are at high risk for developing multi-system acute and chronic conditions associated with significant morbidity and mortality (Mainous et al., 2019; NHLBI, 2014). They can also develop the usual medical comorbidities seen in the general population. Therefore, health care
for adults with SCD should consist of frequent routine visits with a sickle cell expert to monitor for end-organ damage and develop an individualized plan of care for pain and overall disease management and routine preventative care visits with PCPs, dentists, and obstetrician/gynecologists. Primary care should encompass health promotion, disease prevention, health maintenance counseling, patient education, and diagnosis and treatment of acute and chronic illness in a variety of health care settings supported by different health care personnel (IOM, 1996). The PCP’s role is to advocate for the patient within the health care system to accomplish cost-effective care by coordinating services and promoting communication that encourages patients to be fully engaged as active partners (IOM, 1996).
While most children with SCD are cared for by specialists (e.g., pediatric hematologists), most adults transition to PCPs due to the lack of a comprehensive nationwide network of adult SCD providers (Grosse et al., 2009). A recent analysis of data on 1,147 adults with SCD from eight health systems in Florida found that 30.4 percent were cared for by a PCP, while 18.7 percent were cared for by an adult hematologist, 27.5 percent by both a PCP and a hematologist, and 23.3 percent by neither a PCP nor a hematologist (Mainous et al., 2019). Individuals receiving care from both a PCP and a hematologist were less likely to have frequent hospitalizations than those cared for by a single specialist, leading the researchers to conclude that individuals with SCD will benefit from care from both a hematologist and PCP. It is important to establish management strategies between primary care and adult or pediatric sickle cell subspecialists to enhance PCPs’ knowledge of overall SCD care and improve its outcomes.
There are various reasons why very few adults with SCD receive appropriate care in the primary care setting. The challenges of dealing with chronic pain as well as managing opioid therapy, implicit bias and stigma, assumptions around opioid use in SCD reflecting addiction, and provider knowledge deficits about SCD all contribute to reduced access to evidence-based primary care services (Brennan-Cook et al., 2018; Gomes et al., 2015). Many adults with SCD do not consider their PCP as a gateway for accessing more specialized health care services and, therefore, do not make use of specialized services even when they are available. PCPs see a myriad of rare disorders and have significant challenges maintaining expertise and keeping abreast of improvements and new treatments for managing complex conditions (Mehta et al., 2006). A survey of more than 1,000 family physicians found that only 15.7 percent of physicians under the age of 50 and 25.1 percent of physicians over the age of 50 felt comfortable treating individuals with SCD (Mainous et al., 2015). In another study, among 1,288 general internists and pediatricians surveyed, only 32 percent of general internists reported being comfortable providing primary care for adults with SCD (Okumura et al., 2008).
In response to the poor rates of co-management between PCPs and hematologists, one state has established a management model to optimize SCD outcomes and mitigate the high cost of care, particularly for adults. Community Care of North Carolina (CCNC) established a population health model for managing the approximately 3,000 individuals living with SCD in the state in 2017 who were publicly insured under the Medicaid managed care organization. CCNC identified, through multi-stakeholder conversations, barriers to accessing care that led to poor outcomes and increased the cost of care, including irregular/poor follow-up with both PCPs and hematologists, a lack of insurance, and the distance from the specialty SCD centers in the state (Steiner et al., 2008). Through partnerships between the North Carolina Department of Public Health and the six specialty centers, CCNC established a framework to facilitate the implementation of best practices in primary care and co-management between PCPs, specialists, and EDs, leveraging the role of payer-deployed case managers and establishing communication pathways to disseminate provider support tools (CCNC, n.d.; Lunyera et al., 2017; Rushton et al., 2019). The case manager is a member of the health care team who develops a longitudinal relationship with the individuals living with SCD and acquires robust knowledge of their medical experiences and psychosocial and behavioral health needs. The case manager can then tailor care coordination support for each individual based on lived experience to help improve overall QOL and health outcomes (Brennan-Cook et al., 2018).
Implementing clinical decision support tools embedded in the electronic health record (EHR) is another way to support PCPs by providing examples of evidence-based care for individuals living with SCD. One study used a best-practice alert educational information for the provider (implemented in the EHR) about screening for transfusional iron overload using a simple blood test (serum ferritin) (Mainous et al., 2018). Elevated serum ferritin is well established in population studies as increasing the risk for all-cause mortality (Mainous et al., 2004) among adults with SCD (Darbari et al., 2006). Results from one study showed that clinician decision support paired with provider education was particularly effective in helping PCPs address this frequently unrecognized but potentially lethal complication (Peterson et al., 2015). Because the intervention was also cost effective and did not increase the burden on the PCP, it was recommended as a feasible practice change (Peterson et al., 2015).
Like children with SCD, adults with SCD often do not receive the routine preventive care recommended by the U.S. Preventive Services Task Force or the immunizations recommended by the Advisory Committee on Immunization Practices. SCD falls under the category of “additional risk factors,” which includes anatomical or functional asplenia and may include chronic lung, liver, or kidney disease, particularly as adults age and develop
end-organ damage (CDC, 2019b). Asplenia in SCD is often underappreciated by providers and affected individuals. It is also recommended that women of childbearing age regularly use contraception to reduce the risks of unintended pregnancy, with the nuance that the progestin-only and barrier methods are preferred (CDC, 2010; Smith-Whitley, 2014).
There is a significant dearth of high-quality, evidence-based research to guide the ambulatory management of adults with SCD, with even fewer descriptive reports on adult SCD programs (Andemariam and Jones, 2016; Grosse et al., 2009). The lack of evidence poses a challenge for managing these individuals outside of SCD centers. Access to high-quality care is limited further by the limited supply of providers with specific SCD expertise, even within specialty care centers. The committee loosely defines an adult SCD expert as a clinician with both the necessary willingness and prerequisite experience. Unlike in pediatric care, where SCD management is a core component of hematology/oncology fellowship training, an adult SCD specialist may or may not have done a fellowship to become a trained hematologists/oncologist. Many adult hematologists and oncologists are also uncomfortable managing adults with SCD or may prefer to practice oncology, which is perceived to be more lucrative than hematology.
Most adults with SCD will have acute pain episodes, and many more will experience chronic pain, the management of which is nuanced, experiential, and unstandardized. In an ongoing prospective study, Examining Sickle Cell Acute Pain in the Emergency Versus Day Hospital, which examines baseline characteristics of adults with SCD within 60 miles of four cities in the United States, 54 percent of individuals with hemoglobin SS disease and 46 percent with hemoglobin SC disease had three or more acute visits over a 12-month period, and 68 percent of the study cohort reported having chronic pain (Lanzkron et al., 2018a). Therefore, SCD specialists must develop expertise with acute and chronic pain in the context of SCD, a skill that is not often acquired during subspecialty training but rather gained by immersive experience. As will be addressed in Chapter 6 on workforce development, the pool of adult SCD experts in the United States has a diverse training background, including hematology, oncology, pulmonology, critical care, emergency medicine, internal medicine, family medicine, pediatrics, pediatric hematology/oncology, and psychiatry.
8 Due to the lack of documentation of these workforce issues in the literature, some of the discussion in this section is informed by the committee’s expert opinion as practitioners and presentations by invited speakers at committee open session meetings.
Unlike pediatrics, the majority of health care use by adults living with SCD is acute; this reflects the decline in ambulatory care monitoring, especially around the time of transition from pediatric to adult care (Blinder et al., 2015). Medicaid claims data from 8 states on 3,208 individuals showed that access to targeted disease therapies (chronic transfusions, iron chelation, and hydroxyurea [HU]) significantly dropped after age 16, with increased rates of complications and increased health care costs (Blinder et al., 2013).
Ambulatory care for adults with SCD is often not shared or coordinated among providers with the appropriate expertise to manage both SCD and non-SCD comorbidities. A shared care model in which the PCP comanages a patient with complex specialized medical needs alongside a specialist with expertise in the condition of interest has been proposed for adults with SCD, like the models described for children above (Treadwell et al., 2011). This model was evaluated across eight health systems in Florida, and the results suggest that there is a benefit in reducing acute care use; in particular, the model appears to better address total health care needs because individuals benefit from the complementary expertise of the providers (Mainous et al., 2019). This model has been well received, and it is believed to better capture comorbidities and prevent complications (Smith et al., 2008a), leading to successful clinical outcomes among patients with cancer (Klabunde et al., 2013), chronic kidney disease (Scherpbier-de Haan et al., 2013), and mental health issues (Lester, 2005), and it would likely produce similar results for patients with other chronic diseases such as SCD. Research evaluating the comparative effectiveness of various models of co-management will be essential to ensuring improved outcomes for individuals with SCD, particularly as they transition from pediatric to adult care (Grosse et al., 2009).
There is a lack of standardization and clear descriptions of what should be done at each specialty clinic visit and how frequent these visits should be for adults living with SCD. Current trends still assume that “milder” forms of SCD require less frequent medical touch points, despite evidence that these genotypes (SC, SB+ thalassemia) suffer similar burdens of pain and specific organ comorbidities (avascular necrosis, retinopathy) that require more frequent monitoring and attention. Outpatient follow-up with a provider shortly following an acute care visit has been associated with reduced rates of rehospitalization (Leschke et al., 2012).
Missed appointments are a significant challenge and another barrier to care; patients can be dismissed from care if they miss more than two to three appointments with a specific provider (Cronin et al., 2019). It is important that the care team identifies and proactively addresses risk factors for poor adherence to both scheduled appointments and prescribed therapies. These risk factors include social determinants, psychosocial variables, social support, health literacy, and spirituality (Cronin et al., 2018).
Adults with SCD need access to various subspecialists to support the management of their multi-organ comorbidities that are specific to SCD. The most common subspecialists are radiologists, pharmacists, ophthalmologists, orthopedics, obstetrician/gynecologists, pulmonologists, cardiologists, nephrologists, and surgeons (see Table 6-1 in Chapter 6). The committee found no literature on the rates at which adults with SCD are seen by these subspecialty providers, nor did the committee find any literature assessing the experiences of subspecialists in the care of adults with SCD; these areas represent a significant research gap.
Acute pain is the most common reason that individuals with SCD seek health care, even though the vast majority of acute pain episodes are managed at home (Dampier et al., 2002b; McClish et al., 2006; Smith et al., 2008b). Such episodes are particularly an issue for adults (Yale et al., 2000). Children and adolescents also experience acute pain episodes. For example, one study conducted in children and adolescents, which required participants to keep daily diary entries to record their pain history, found that daily pain and acute pain exacerbations are relatively infrequent in childhood but increase in adolescence (Dampier et al., 2002a).
There are long-term consequences associated with poorly treated acute pain in individuals with SCD, including progression to chronic pain syndromes, adverse effects of chronic opioid usage, psychological maladjustment, poor QOL, and excessive use of health care (Telfer and Kaya, 2017). When acute pain is left untreated, there is the potential for acute neurohumoral changes, neuronal remodeling, and long-lasting psychological, emotional, and economic distress, which may ultimately lead to prolonged chronic pain states (Dunwoody et al., 2008; Gjeilo et al., 2014; Polomano et al., 2008).
Regardless of the approach, adequately managing acute pain is the only known way to prevent the development of chronic pain, which has been well described and appreciated in the literature concerning post-operative pain. Nevertheless, this method to address acute pain has been poorly applied in the SCD context, even though SCD has a well-known pathophysiological mechanism that results in recurrent episodes of acute pain. Unfortunately, for nearly a century these consequences of poorly treated acute pain have not been considered in the development of standardized home-based SCD pain management protocols, nor have they been considered in the education and communication about SCD pain and its management for patients and providers. There remains a major research gap in this area that has been heightened by the emotional response of providers and the community to the widely publicized opioid crisis.
Home-Based SCD Pain Management
Currently, the home management of acute SCD pain in adults typically starts with physical rest and oral hydration, followed by as-needed oral analgesics with or without muscle relaxants. The oral analgesics include acetaminophen, non-steroidal anti-inflammatory drugs (NSAIDs), and various opioid preparations. Many adults, however, are on daily, long-acting opioids for chronic pain and are advised to continue taking these and to add short-acting preparations for pain management. Ideally, this regimen would be developed using shared decision making with the SCD provider to establish an individualized pain management plan (Ballas, 2005; Balsamo et al., 2019).
For adults with SCD, chronic pain is often the norm rather than the exception. One study found that 51 percent of participants reported pain on at least half of the days during the study period (Smith et al., 2008b). Any pain was 10 times more likely to be managed at home than in the acute care setting, whereas crisis pain was only 4 times more likely to be managed at home (Smith et al., 2008b). Interestingly, home opioid use was variable and was related to having more pain, more crises, and higher acute care use. This offers a broad research opportunity to help individuals with SCD establish a structured regimen for managing their various types of pain (e.g., chronic pain, acute pain exacerbation, acute pain crisis) logically and systematically, using opioids when appropriate (Smith et al., 2008b).
Systematic efforts to help individuals living with SCD understand their personal pain experience and adopt optimal evidence-based strategies for acute SCD pain self-management are scarce, perhaps because of the difficulty these individuals encounter in describing their pain (Jenerette et al., 2014; Matthie and Jenerette, 2017). Especially starting in adolescence, when pain experiences start to peak, individuals report having significant barriers to effectively describing and characterizing their pain (Lee et al., 2012). Individuals may also seek pain relief through complementary and alternative methods, such as prayer, relaxation techniques, massage, exercise, and spiritual healing; however, these methods have not been well studied and are not well understood by the health care establishment (Clayton-Jones and Haglund, 2016; Thompson and Eriator, 2014). While the research on the efficacy of these approaches is limited, providers need to be aware of them in developing patient-centered approaches for care management (Mongiovi et al., 2016).
Institutional Acute Care
Home-based pain management and health system–based acute SCD pain management (acute care use) occur at the opposite ends of a wide spectrum of SCD pain frequency and severity (Smith et al., 2008b). When
pain management is attempted at home and fails, individuals will seek acute care. Acute SCD pain that requires health care use is addressed either in the ED or in a separate hospital-based location designed to avoid the delays in access to pain management that are often inherent in busy urban EDs. If the pain is not controlled at home, patients may elect to go to a day infusion center, an ED, or a hospital clinical decision unit (CDU). Chapter 2 detailed some of the challenges for getting care even in the ED.
Day Hospitals and Infusion Centers
There are several day hospitals or infusion centers around the country, some of which are solely for the use of adults presenting with acute vasoocclusive crisis (VOC) and others that function as shared facilities for SCD and other hematological diagnoses. These facilities are commonly available for limited times and provide analgesic and supportive therapy for those not requiring an extended stay to manage an uncomplicated acute SCD pain episode (Lanzkron et al., 2015). This approach to care is resource intensive and requires dedicated physical space along with specialty-trained multidisciplinary personnel. However, these facilities offer a viable strategy for managing acute VOC pain due to their effectiveness in reducing admission rates and length of stay and overall health care costs compared with care in the ED (Adewoye et al., 2007; Benjamin et al., 2000; Han et al., 2018).
Effective treatment of an acute SCD pain episode follows three main principles: (1) provide prompt pain control using analgesics with high bioavailability; (2) use supportive care strategies that include intracellular re-hydration with hypotonic fluid (oral or intravenous), correction of hypoxia and acidosis, and other supportive care, including rest; and (3) use targeted treatment of the underlying trigger, such as infection or other complications (Platt et al., 2002). Often the third component is overlooked, not investigated, or ignored, and individuals with SCD suffer needless progression of complications and high morbidity and mortality. The Georgia Comprehensive Sickle Cell Center established the first 24-hour acute care center for SCD with a philosophy of prompt access to pain relief and engagement in ongoing comprehensive care to improve disease outcomes for its large SCD population (see Appendix F). There are algorithms for treating an acute pain episode using a combination of opioids and NSAIDs, with guidance on optimal dosing based on the half-life of each medication and frequent close monitoring for response and toxicity (Platt et al., 2002; Raphael et al., 2008). According to these recommendations, oral or parenteral analgesia should be supplied as promptly as possible, with current guidelines recommending within 30 minutes of triage or within 60 minutes of registration (NHLBI, 2014). This promptness is now considered a measure of quality of care for SCD. It is appropriate to re-evaluate the individual after the
first dose of analgesia. This evaluation is important in determining the efficacy of the treatment and in identifying any untoward toxicity, such as the oversedation, itching, and nausea that commonly occur with opiates. A frequent objective reassessment of the individual for pain and other symptoms is recommended every 15 to 30 minutes, with redosing of the analgesic in the case of poor pain control. These actions are associated with fewer hospital admissions, reduced length of stay, and increased patient satisfaction (Brandow et al., 2016; Inoue et al., 2016; Kavanagh et al., 2015; Tanabe et al., 2017).
Unfortunately, while re-evaluating pain status and redosing analgesia within the recommended 15–30 minutes may occur in an SCD day hospital or CDU, as discussed below, it is rare in most EDs, for variety of reasons. Additional evidence-based strategies for optimizing ED acute pain management are needed (Glassberg, 2017).
Clinical Decision Unit
A CDU is a newer care option for evaluating and treating acute VOC pain. It allows for the evaluation of pain and high-dose analgesic therapy over an extended period in an inpatient-like setting. CDUs are typically open for longer periods than infusion centers but follow similar protocol-based algorithms for delivering optimal pain management and investigating the reasons or triggers for acute pain exacerbation.
While CDUs are not dedicated to SCD patients (they address a wide variety of illnesses for which patients may require up to 24 hours of care), uncomplicated VOC is a diagnosis that, with proper entry criteria and an individualized treatment plan, can be successfully treated with a short stay in a CDU (Cline et al., 2018). There are robust data on the pharmacoeconomic and clinical superiority of managing acute VOC using these specialized/targeted facilities with the right staffing and infrastructure.
A recent study comparing the care received by 370 children seeking care for acute VOC in either a CDU or the ED showed that the 140 children with SCD who were managed in the CDU were given the choice of an initial analgesic that was adherent to pain management guidelines (84 percent versus 45 percent), had less time to first analgesia (32 minutes versus 70 minutes), and had a lower admission rate (29 percent versus 57 percent [odds ratio (OR) = 3.82; 95% CI 1.87–7.82]) than children receiving care in the ED (Karkoska et al., 2019). While an ED-based dedicated observation unit has strong potential to affect the quality of the pain treatment, individuals with SCD continue to report negative experiences with provider attitudes, implicit bias, and stigma during their encounters with the ED and acute care in general. The committee was unable to find studies that investigated CDUs for adults, but there is no reason to assume it would not also be effective for adults living with SCD.
The ED is the most commonly used resource to evaluate and treat acute VOC pain and SCD complications in adults. In eight geographically disparate states in the United States containing 21,112 individuals with SCD, there was a mean of 2.59 acute care encounters per person per year, with the highest rates observed among publicly insured 18- to 30-year-olds (4.8 acute care encounters per person year) (Brousseau et al., 2010). EDs are open 24 hours per day, 7 days per week and have the resources to provide comprehensive evaluation and treatment. The ED has the advantage of being able to provide parenteral analgesic that may not available in the outpatient setting.
In addition, the ED is the optimal place to address unusual pain presentations or additional symptoms, such as shortness of breath and mental status changes that may herald a severe complication rather than an uncomplicated pain crisis (Telfer and Kaya, 2017). For a person who does not have readily available transportation to a CDU or SCD day hospital, access to the ED is often possible by calling 911. Thus, it is possible that individuals with SCD whose health care needs could be managed at a CDU or SCD day hospital may end up in the ED. The disadvantage of seeking care for acute SCD pain in the ED is the lack of familiarity between patient and provider, delayed care due to ED overcrowding, and the lack of provider familiarity with SCD. ED care is also quite expensive. According to 2009 estimates for approximately 70,000 individuals with SCD in the United States, the cumulative health care costs for individuals living with SCD exceed $1.1 billion per annum, representing mostly acute care use or ED and hospital admissions (Kauf et al., 2009). There is a nationwide trend to implement ED observational units to manage overcrowding and contain costs (Wiler et al., 2011). To standardize acute SCD pain management and optimize care outcomes in EDs, some EDs have implemented an observation unit as an alternative to a separate CDU or day hospital (Gowhari et al., 2015; Lyon et al., 2014). Regardless of the location of care, providers and health care teams should follow the tenets outlined in Table 5-1.
One major issue concerning individuals, particularly adults, with SCD is the extensive use of EDs for acute pain management. Paulukonis et al. (2017) performed the largest epidemiologic study on ED use by SCD patients, following a cohort of 4,636 California SCD patients over a 10-year period (2005–2014). The study found that nearly all (93 percent) of those individuals had visited an ED at least once (for any diagnosis) and 4,100 (88 percent) of the study participants had at least one treat-and-release ED encounter during that time (Paulukonis et al., 2017). The average number of ED visits was 2.1 visits per person per year. In a single year
TABLE 5-1 The ABCs for Managing Acute Sickle Cell Pain
|A||Assess pain using a universal pain assessment tool.|
|B||Believe the patient’s pain level.|
|C||Look for complications and causes, such as infection, gallstones, and splenomegaly.|
Implement drugs and distraction, using
|E||Ensure that the environment is conducive to rest. The area should be quiet, with privacy.|
|F||Provide fluids (hypotonic D5W or D5 1/4 NS). Give a fixed dosing of analgesia—on a time schedule, not as needed or PRN.|
NOTE: NHLBI = National Heart, Lung, and Blood Institute; NSAID = non-steroidal anti-inflammatory drug; PRN = pro re nata (“when necessary”).
SOURCE: Adapted from Platt et al., 2002, with permission.
(2005), 53 percent of those in the cohort had no treat-and-release ED visits, 35 percent had 1–3 visits, 9 percent had 4–10 visits, and 3 percent had 11 or more visits; individuals in the highest-use group accounted for 45 percent of all of the ED visits during the study period. ED use was highest among young adults and higher among older adults than children (Paulukonis et al., 2017). Other studies have estimated an acute care use rate of 2.59 to 3.0 visits per patient per year (Brousseau et al., 2010; Lanzkron et al., 2018a). In two studies, adults accounted for the majority of all ED visits and hospitalizations (Lanzkron et al., 2010; Yusuf et al., 2010).
The information the committee found regarding ED visits by patients with SCD is the result of analyses of data collected from either a single clinical institution, a consortium of institutions, or a dataset based on a single source of administrative data (i.e., hospital discharge or Medicaid); each dataset has strengths and weaknesses associated with data quality. Data solely from clinical care centers may not accurately reflect the general SCD population. By contrast, administrative data rely on International Classification of Diseases (ICD) codes to identify patients with SCD, and these data include a larger number of patients, including those who receive care outside of SCD clinical centers. However, the correlation between SCD ICD codes and a laboratory-confirmed diagnosis of SCD has been deemed unsatisfactory (Paulukonis et al., 2017). Thus, larger-scale epidemiologic studies are needed to develop an accurate measure of acute care use by SCD patients to guide treatment protocols (Lanzkron et al., 2015).
Pain is one of the most common reasons for hospitalization and readmission in individuals with SCD (Ho et al., 2019); however, the complications leading to readmission may also include pain and fever. Annual hospitalization rates for patients with SCD are 178 to 216 per 100,000 (Okam et al., 2014). Hospitalization rates did not decrease after the U.S. Food and Drug Administration approval of HU (Okam et al., 2014). One possible explanation for this may be limited access to or poor adherence to HU, per a study conducted in adolescents (Badawy et al., 2018b).
As noted in Chapter 4, SCD is one of the 10 most common reasons for pediatric hospital readmissions (Heslin et al., 2018). Readmission rates for adults with SCD are high as well. Risk factors for readmission vary, but lack of a PCP and a recent missed appointment are important indicators in the adult population (Brodsky et al., 2017; Cronin et al., 2019).
Unplanned readmission rates were highest for 18- to 30-year-olds (Brousseau et al., 2010). Readmission is also higher among those with public insurance (Brousseau et al., 2010). The percentage of readmissions that are medically necessary versus those that are not has not been determined.
High-Use Acute Care
A small subpopulation of individuals living with SCD accesses acute care at rates significantly more than two standard deviations above the mean of the population. These individuals are often referred to as “super-users” and may present to sickle cell day units and hospital EDs much more frequently than others with a similar disease and comorbidity burden (Carroll et al., 2011). One study found that approximately 20 percent of the SCD population accounts for 54 percent of all ED visits (Epstein et al., 2006).
Super-users may have a more severe level of disease, have a higher psychosocial and behavioral health burden with less social support, or lack coordinated care resources (Aisiku et al., 2009). While the data are limited, Simpson et al. (2017) found that instituting comprehensive coordinated health care plans involving a multitude of support services (physicians, advanced practice providers, pharmacists, social workers, psychologists, and psychiatrists) was beneficial for a small sample of super-user adults. The intervention was also beneficial in reducing annualized acute care use across all measured indexes (Simpson et al., 2017). Compared with preintervention, the authors found statistically significant reductions in the annualized number of ED visits (decrease of 16.5 visits per patient-year). They also found lower annualized ED length of stay (decreased by 115.3 hours per patient-year), inpatient admissions (decreased by 4.20 admissions per patient-year), inpatient length of stay (decreased by 35.8 hours
per patient-year), and visits where the patient left before treatment (decreased by 13.7 visits per patient-year) (Simpson et al., 2017).
The transition from inpatient hospitalization or ED visits to home provides an opportunity to decrease hospital readmission or return visits to the ED. The transitional care strategies from the hospital to home for patients with SCD during this process are not reported. This may represent a missed opportunity for case management by nurses and CHWs (Logan, 2019).
A novel program, Aiming to Improve Readmissions Through Integrated Hospital Transitions, was established by a regional health care system to smooth the transition from acute care to community residence by reducing the 30-day readmission rate for a population with a high risk for readmission (McWilliams et al., 2019). This unblinded pragmatic randomized controlled trial included 1,876 patients under the care of a hospitalist for their inpatient admission to receive either usual care after discharge or care in a dedicated post-acute care multidisciplinary transition services clinic run by hospitalists. The clinic offered close follow-up for 30 days with access to a free-standing clinic that included a transition-dedicated internist, a pharmacist, and paramedicine, behavioral health, and social work providers, with scheduling frequency and provider type matched to patient needs (McWilliams et al., 2019). The hospital follow-up could occur either in person or virtually in patients’ homes (facilitated by paramedicine) and included a comprehensive medication reconciliation by a pharmacist and, at a minimum, weekly contact with a transition services team member with coordinated support to transition to the next appropriate care location after 30 days. While the 30-day readmission rate did not change in the group receiving transition services, those readmitted were less likely to require intensive care compared with the usual care group (15.5 percent versus 26.8 percent) (p < 0.02) (McWilliams et al., 2019). These transition services were later made available to adults with SCD as a non-randomized cohort with targeted efforts to increase community acceptance and engagement and strong collaboration between the SCD provider and the transition services provider team. Early indications show that there has been a positive impact on reducing readmission rates for adults with SCD who use transition services.
Rehabilitation services are needed for multiple reasons in the SCD population. Physical disability (Swanson et al., 2011) is not uncommon and occurs after complications, such as stroke, avascular necrosis of the hips and shoulders, and leg ulcers, or after a prolonged hospitalization
with extremely limited physical activity, such as those hospitalizations that occur with pain episodes. Historically, physical medicine and rehabilitation inpatient stays occurred after stroke in order to address the motor deficits associated with cerebral infarction and hemorrhage. For many children and adults with SCD and avascular necrosis of the hips and shoulders, physical and occupational therapy are used to improve pain and address mobility after surgical interventions. Patients with shoulder avascular necrosis require occupational therapy, but their use of these services is under-reported. Many require surgical management of avascular necrosis, which includes core decompression and hip replacement (Mallet et al., 2018). Physical therapy after hip replacement is essential for recovery, but where these services occur varies widely in the general population. Inpatient and outpatient services to improve mobility after surgery are limited by access to local services, transportation, and insurance (Clarke et al., 2017). The use of these services needs to be addressed in children and adults with SCD.
Kinesiotherapy, aquatic rehabilitation, and physical therapy may all be effective for managing acute and chronic pain (Alcorn et al., 1984). The use of physical therapy is not reported but seems to be prescribed particularly during or following prolonged inpatient stays for pain.
Palliative care services are a holistic approach that includes advance care planning, disease management, pain relief, and support for bereavement and grief using open and supportive communication strategies among the health care team, patients, and their families (Wilkie et al., 2010). Palliative care services to improve the QOL for people with serious illness, such as SCD, have been explored as a method to improve pain management and to support end of life, which typically occurs two to three decades earlier in individuals with SCD than in the general population (Ajayi et al., 2016). More than 78 percent of adults with SCD die in the ED or hospital at an average age of 45 (±16 years).
Palliative services are, however, underused during end-of-life care for individuals with SCD (Johnston et al., 2019). Referral patterns for palliative care for non-cancer patients often reflect the need for support with symptom management, particularly for pain (Ghanem et al., 2011). The factors influencing patterns of palliative care service use require further exploration.
Transfusion Medicine Services
Children and adults with SCD require intermittent and long-term red blood cell (RBC) transfusions across the life span to decrease the proportion of sickle hemoglobin (HbS) relative to hemoglobin A and to increase the
oxygen-carrying capacity of RBCs, as discussed in Chapter 4. Transfusions may be administered as simple or exchange transfusions. Exchange transfusions may be manual or automated, both of which are equally effective, but patients and providers may prefer the ease of automated exchange. However, access to automated exchange transfusion may be limited in some hospitals and in the outpatient setting for several reasons, including equipment cost and maintenance and prior inability to bill at an increased level for the procedure.
Published data on behavioral or mental health service use in children and adults with SCD suggest that children with mental health conditions use health services–related pain management at higher rates than those without such conditions, citing higher annual admission rates and longer lengths of stay (Myrvik et al., 2013) (see Chapter 4 for a more detailed discussion on behavioral and mental health). However, other studies imply that poor physical functioning rather than poor mental functioning increases health care use in adults with SCD (Artz et al., 2009). Few data are published on health care use that does not involve pain management. However, in other chronic illnesses, such as obesity, adults with both obesity and mental illness use health services more than those with just obesity (Shen et al., 2008).
Facilitating Employment for Individuals Living with SCD
The Office of Disability Employment Policy in the U.S. Department of Labor offers guidelines for workplace accommodations for individuals living with SCD (JAN, 2019). Eligibility for accommodations is outlined under the Americans with Disabilities Act. These accommodations may include allowing for a flexible schedule in order for the individual to receive necessary medical treatment, the provision of an adjustable workstation, and the use of an aide if needed.
Facilitating employment for individuals living with SCD can be a positive factor in managing health care use. Williams et al. (2018) followed 95 individuals living with SCD prospectively and found that having employment was significantly associated with decreased health care interactions. More information about employment outcomes for individuals living with SCD can be found in Chapter 2.
COMPREHENSIVE SCD CARE DELIVERY MODEL
Ultimately, enabling the delivery of effective treatment to all individuals living with SCD at all stages of the disease will require developing and
implementing a comprehensive SCD care delivery model. There have been efforts toward this end, but the resulting models fall short of ideal in various ways. This section examines the current state of comprehensive SCD care, its strengths and weaknesses, and systems of comprehensive care for other diseases that could serve as models for SCD.
Overview of Historical, Federally Funded SCD Comprehensive Care Centers
Historically, the terminology “comprehensive sickle cell centers” was applied to those receiving funding through the National Institutes of Health’s comprehensive sickle cell centers program from 1972 to 2013 (NHLBI, 2006). These programs were funded for SCD basic, translational, and clinical research and had strong clinical services at the core by providing activities such as diagnosis, counseling, and education concerning SCD and related disorders. This philosophy of strong clinical care as the backbone of research initiatives stemmed from the early comprehensive sickle cell centers, created by the 1972 National Sickle Cell Anemia Control Act, which provided the authority to establish treatment programs. Funding was limited, and no more than 10 centers were funded (Howard University, n.d.). When federal support for these centers ended, many sickle cell programs that were not federally funded had adopted the terminology “comprehensive sickle cell center” for their clinical care programs but without the assurance that they had all the required components.
Although there is little published on the history of the comprehensive sickle cell centers, their influence set the stage for the infrastructure of sickle cell programs. In 2008 the National Heart, Lung, and Blood Institute research program was restructured to focus on basic and translational research, specific grants, an expansion of the trials network to attract a broader array of participants, enhanced genomic research, and guidelines (NHLBI, 2008). This shift also led to the genesis of the concept of a clinical “center” for the care of children and adults with SCD that was termed the “comprehensive sickle cell center.”
Defining a Model for Comprehensive SCD Care
As with many rare conditions with early childhood mortality, the focus of SCD care in the 1960s and 1970s was on curtailing mortality by preventing infections. The splenic dysfunction that leads to an increased risk for infection was well established by the time that the 1972 Sickle Cell Disease Control Act was implemented. One of the first efforts of the comprehensive sickle cell centers was the Prophylactic Penicillin Study in 1983 (Gaston et al., 1986). This randomized controlled trial demonstrated an 84 percent
decreased rate of infection in the penicillin group (Gaston et al., 1986). Initiating oral penicillin early in life, before splenic dysfunction, became the goal for a national newborn screening (NBS) program (Therrell et al., 2015).
The success of the NBS program and improved access to comprehensive care for children with SCD increased the proportion of children living to their third decade (Quinn et al., 2010). Comprehensive care was bolstered by implementing evidence-based care for infection and stroke prevention and by fostering widespread use of HU and protocol-driven pediatric care. Unfortunately, the resources adopted to support pediatric care did not evolve to include the adult care setting. Currently, sickle cell experts for adult-focused care are in short supply but increasingly high demand (see Chapter 6).
Key components of “comprehensive sickle cell centers” include a system of care for children and adults with SCD across the life span. Services include health maintenance and preventive care in outpatient settings, where patient education, anticipatory guidance, and behavioral health are managed by a multidisciplinary team. Individuals with SCD have psychosocial needs that should be addressed along with their medical needs. Centers often understand patients’ challenges relating to stigma and health disparities, although this was not mandated in the funding requirements. These centers were embedded within health care systems that provided for the unique health care needs of SCD and were geared to ensure coordinated care. Below is a brief description of the desirable components, identified by the committee, of a team-based, comprehensive care delivery model:
- Health maintenance and prevention. These are services aimed at establishing a disease steady state across the life span, including genetic testing for hemoglobin variants and RBC antigens and the management of long-term therapies, including HU, chronic transfusions, and stem cell therapies.
- Medical subspecialty care. This was initially focused on support for stroke and neurocognitive disorder management and pulmonology for asthma, ACS, and chronic lung disease management. It has since evolved to include a myriad of specialists targeting the multi-system complications of SCD discussed in Chapter 4.
- Neuropsychiatric and neurology services. These services include evaluation for neurocognitive deficits, executive function, cerebral ischemia (“silent” and “overt” manifestations), and cerebral vasculopathy. Neuroimaging to assess Chiari malformations and aneurysms is a component of care, coupled with radiology services.
- Behavioral health. These services address depression, posttraumatic stress, anxiety, pica, opioid use disorders, and other behavioral health issues in an environment of care that acknowledges poverty, racism, and its associated behavioral health management strategies.
- Radiology. These are specialized services for TCD ultrasonography for the accurate assessment of TCD velocities to assess stroke risk, echocardiology techniques to accurately assess tricuspid regurgitation jet velocities, imaging scans to assess pulmonary hypertension, magnetic resonance imaging for stroke management, and imaging tests to detect avascular necrosis.
- Blood bank and transfusion medicine. These appropriately provide RBC units with extended antigen matching, the administration of RBC quantities that avoid hyperviscosity, automated and manual erythrocytapheresis to rapidly decrease HbS without increasing overall hemoglobin values, and the minimization of transfusional iron overload.
- Surgical care. These surgical subspecialty services are familiar with common indications for surgery, such as cholecystectomy, splenectomy, hip replacement, adenotonsillectomy, C-section, and ophthalmologic procedures, and the risk of surgical complications unique to SCD peri-operatively and long term.
- Anesthesia and sedation services. These are specialized services to assess risk for the development of post-operative ACS.
- Genetic counseling. These activities provide genetic counseling services to individuals diagnosed by NBS or other screening modalities, with accurate anticipatory guidance provided concerning sickle cell trait (SCT) diagnosis, pregnancy planning, and other issues.
- Education and vocational services. These activities provide training and information about SCD to health care professionals at the center and in the community or region that the center serves and to patients, their families, and communities, with linkages to community support to optimize patient education and vocation.
- Research. The comprehensive sickle cell centers conducted research to improve the treatment and prevention of complications, enhance the transfer of these new findings to the clinical setting, and identify new research directions. While research was a core component of the original design, centers were also required to provide supportive activities in diagnosis, management education, and counseling and to spread the research agenda across basic, translational, clinical, and outcomes topics.
- Data management core. Each center was supported by a central data coordinating point as a shared resource for data management, statistical support, and the standardization and development of research protocols, data element definitions, and staff training.
- Physical and occupational therapy. These are ambulatory services in physical therapy, occupational therapy, and physical medicine and rehabilitation for avascular necrosis, reconditioning following
- prolonged hospitalizations, and weight management for healthy weight.
- Community outreach. This involved establishing communication channels between the comprehensive sickle cell center and the community via liaisons, who would inform the community about programs and provide community input into programmatic development and priorities. Traditionally, these liaisons were members of a formally established SCD CBO with a close affiliation via formal membership to the national advocacy agency (the Sickle Cell Disease Association of America) and a broad scope of supportive services (see Chapter 8 for more information). One step in improving services in the community might be to tap into the experiences of these groups to identify and disseminate best practices.
The comprehensive centers model was an excellent attempt to operationalize a care delivery model for infants and children, who had an exorbitantly high childhood mortality rate at that time. The next iteration of comprehensive centers should include attention to the morbidities that affect the aging population along with formalized tracking and measurement of both the research and clinical and supportive care components. There are various models through which this care may be provided, and the following section outlines one.
Redesigning the Care Delivery Model
After the release of the Institute of Medicine (IOM) report Crossing the Quality Chasm (IOM, 2001b), which highlighted the highly fragmented nature and poorly designed care processes of the American health system, new models for health care delivery were developed and implemented, such as the patient-centered medical home (PCMH) and comprehensive primary care plus (CPC+). These models were designed in response to challenges identified in that report (IOM, 2001b):
- Redesign care processes to effectively meet the needs of the chronically ill.
- Improve information technologies to support chronic care.
- Manage the growing knowledge base with training, workforce development, and clinical decision support.
- Coordinate patient care across time, specialty, and location/intensity of service (outpatient and inpatient).
- Advance effectiveness of teams and multidisciplinary training.
- Incorporate process and outcome measurement into daily work.
The findings from the 2014–2017 SCDTDRCP and its precursor, the Working to Improve Sickle Cell Healthcare project (2011–2015), recommended that developing a system of care for individuals with SCD should include the main tenets of the PCMH such as a PCP for comprehensive care and continuous relationship, a multidisciplinary team, and specialists and community providers (Adams et al., 2017). These recommendations and proposed operational elements have been incorporated into specific care delivery models, such as the PCMH and CPC+. Other disease-specific models, such as the cystic fibrosis (CF) care center accreditation standards, address these recommendations in way that are similar to how they are addressed by the PCMH and CPC+. However, such recommendations should take into consideration that health systems are complex, adaptive, and governed by shared goals and simple rules (IOM, 2001b). The current standard of SCD care in the United States does not follow the new rules for a 21st-century health care system (IOM, 2001b) (see Table 5-2). This will need to be changed in order to deliver team-based comprehensive care to individuals with SCD.
The PCMH is a model for delivering care with a core focus on advancing primary care and the health care home (Rich et al., 2012). The PCMH focuses on comprehensive, patient-centered, coordinated care with accessible services and commitment to quality and safety (Rich et al., 2012). Comprehensive care involves using a team-based approach to provide mental and physical health care. Patient-centered care is oriented around the
TABLE 5-2 Simple Rules for the 21st-Century Health Care System
|Current Approach||New Rule|
|Care is based primarily on visits.||Care is based on a continuous healing relationship.|
|Professional autonomy drives variability.||Care is customized according to patient needs.|
|Professionals control care.||The patient is the source of control.|
|Information is a record.||Knowledge is shared and information flows.|
|Decision making is based on training and experience.||Decision making is evidence based.|
|Do no harm is an individual responsibility.||Safety is a system property.|
|Secrecy is necessary.||Transparency is necessary.|
|The system reacts to needs.||Needs are anticipated.|
|Cost reduction is sought.||Waste is continuously decreased.|
|Preference is given to professional roles over the system.||Cooperation among clinicians is a priority.|
SOURCE: IOM, 2001b.
patient’s needs, values, and preferences. Coordinated care provides the patient with acute and chronic care resources across a continuum. Accessible services include telehealth and after-hour health services to ensure that patients’ needs and preferences are met. Finally, the PCMH’s commitment to quality and safety involves using patient data for quality improvement purposes.
CPC+ is a primary care medical home model with a focus on advancing primary care through regionally based multi-payer payment reform and care delivery transformation (CMS, 2019). By 2019, 2,851 primary care practices and 55 payers across 18 regions in the United States were participating in CPC+ (CMS, 2019). Key functions of CPC+ include access and continuity, care management, comprehensiveness and coordination, patient and caregiver engagement, and planned care and population health. The three payment elements of CPC+ include a care management fee, performance-based incentive payment, and payment under the Medicare physician fee schedule.
The committee reviewed the PCMH, CPC+, CF care centers, and the hemophilia model of care and identified the following examples of how those care models operationalized the structural elements of an ideal care model (see Box 5-1).
Comparison with Cystic Fibrosis
The Cystic Fibrosis Foundation (CFF) accreditation rules maximize the learning of the complex, adaptive, health care system. The lack of these guidelines for SCD leaves care unimproved. While there is more than one method to accomplish good-quality care delivery for SCD, the absence of standards, expectations, and funding creates disparities within health care and between SCD care and that of other rare diseases, such as CF (recognized as one of the best examples of system design for a chronic disease of childhood). At a minimum, a single health professional could coordinate care for a population of patients with SCD, coordinating appointments, providing specialty-specific checklists, maintaining transition registries and disease registries, providing disease-specific education for patients and families, and connecting PCPs and specialty providers (Bodenheimer et al., 2002b).
As a model for excellent outcomes in childhood chronic illness, CFF requires accredited centers to prepare the workforce, with all specialties demonstrating teaching medical students, residents, and other workers in the health care field the highest standards of care. Such a requirement would also help SCD and other rare and childhood chronic illnesses. Additionally, educational materials and even “certification” pathways for health professionals to become proficient in SCD management would be a mechanism
for increasing payment for practices that perform population management and high-quality care across all domains of care.
Comparison with Hemophilia
Like SCD and CF, hemophilia is a rare hereditary disorder whose treatment uses significant resources for the health care system (Tarantino
and Pindolia, 2017). In 2016 the National Hemophilia Foundation in collaboration with McMaster University created a guideline on the use of care models for managing hemophilia. This guidance proposes the use of an integrated care model, including an integrated care team (NHF, 2016).
The evidence on the impact of the integrated care model for the hemophilia population is limited but positive; two separate research reviews cited the lack of rigorous research on the impact of hemophilia care models. Young et al. (2016) conducted a systematic review and found some evidence (described as low- to very low-quality) that, in comparison with other models of care, the integrated care model for hemophilia produces better outcomes (decreased mortality and fewer hospitalizations, fewer ED visits, fewer missed school and work days). Stoffman et al. (2019) reviewed research regarding hemophilia care according to the World Federation of Hemophilia guidelines and described four aspects of care models that are recommended: global guidelines and protocols, collaboration with other countries, patient registries to track resource use, and the provision of personal care to optimize treatment outcomes. The CF model and hemophilia are further described in Chapter 8.
Sickle Cell Disease Treatment Demonstration Regional Collaborative Program
The SCDTDRCP, which began in 2004 and was funded by HRSA, serves as a good model for organizing the delivery of comprehensive SCD care. Currently the SCDTDRCP provides grants to five regional networks consisting of sickle cell treatment centers, federally qualified health centers, and CBOs in 43 states across the United States to provide coordinated, comprehensive, culturally competent, and family-centered care to people with SCD and SCT9 (Adams et al., 2017). The goals of the SCDTDRCP are to improve care delivery and access for people with SCD and SCT, increase the number of providers with SCD expertise and knowledge of SCD treatment methods, and increase the use of HU for people with SCD. To improve access to medical care and educational services, teams in the SCDTDRCP have implemented Project Extension for Community Healthcare Outcomes (ECHO), which uses telementoring to connect local clinicians to experts from sickle cell centers and allows hematologists to share knowledge with PCPs via real-time, virtual provider-to-provider education and mentoring for sickle cell care and webinars on relevant SCD topics (Adams et al.,
9 The five regional grantees in the SCDTDRCP are Johns Hopkins in the Northeast region, Charlotte Mecklenburg Hospital in the Southeast region, Cincinnati Children’s Hospital in the Midwest region, Washington University in the Heartland and Southwest region, and the Center for Inherited Blood Disorders in the Pacific region (Adams et al., 2017).
2017). The SCDTDRCP also improves the patient–provider interaction by using the Chronic Care Model (CCM) approach. The goal of the CCM is to improve health outcomes for patients with chronic illnesses by changing care delivery (Bodenheimer et al., 2002a). In order to provide patient-centered, evidence-based care, the CCM focuses on six components: community resources and policies, health care organization, self-management support, delivery system design, decision support, and clinical information systems (Bodenheimer et al., 2002a). The SCDTDRCP also offers a framework for continuous improvement; information collected from the SCDTDRCP, through a national collaborative website, was analyzed using core metrics to identify best practices for care delivery, educational materials for SCD treatment, and the efficacy of the SCDTDRCP (Adams et al., 2017). An evaluation of the SCDTDRCP showed demonstrable results in improving access to care, HU use, and provider education (see Appendix H). The geographic organization and reach of the program as well as the existing networks could form the basis of comprehensive models of care throughout the country.
Hemoglobinopathy Learning Collaborative
The Hemoglobinopathy Learning Collaborative, which was created under the auspices of the SCDTDRCP, has been implementing continuous quality improvement principles in the management and outcomes of SCD and other hemoglobinopathies (Oyeku et al., 2012). In a Delphi study, five drivers of quality improvement were identified: a strong community network; knowledgeable individuals, families, and providers; reliable identification and follow-up; seamless co-management between primary and specialty care; and the appropriate treatment of acute episodes (Oyeku et al., 2012). These five themes are consistent with PCMH, CPC+, and CF structural elements and should be included in SCD care.
BARRIERS TO COMPREHENSIVE CARE
There are various barriers to developing a system of comprehensive care for SCD; this section describes several such barriers and possible ways to overcome them.
Few individuals living with SCD are seen by or have access to a comprehensive sickle cell center with specialized providers (Grosse et al., 2009). One barrier to comprehensive care for the chronically ill is geographic distance from specialized care, especially in rural areas (Kimmel et al., 2018).
In addition, individuals with SCD may have difficulty finding appropriate primary care for several reasons, including the discomfort of providers with ambulatory care or managing disease-specific conditions (Whiteman et al., 2015). A lack of familiarity with SCD may also result in clinician attitudes that further limit care (Haywood et al., 2009). The CDC Sickle Cell Data Collection Project is collecting data on the geographic location of the sickle cell population in California and Georgia. This information combined with health care usage data will inform unmet health needs. Models such as Project ECHO should be explored to improve provider access to sickle cell expertise to improve patient outcomes (Zhou et al., 2016).
Innovative Care Models to Overcome Geographic Barriers
To overcome geographic barriers, newly available technologies may prove useful. These innovative approaches may focus on the individual with SCD and on providers. E-mail and text messaging have allowed patients far greater ease of connecting with providers (Raphael and Oyeku, 2013). For providers, telemedicine with videos has been used to enhance the delivery of primary care (Woods et al., 1998). A more sophisticated approach, Project ECHO, is an interactive model of training hospital and acute care providers in high-quality, culturally sensitive care for individuals with SCD (Arora et al., 2007). Project ECHO uses telementoring through a hub-and-spoke model, in which the expert providers are the academic “hub” and the clinicians in the local communities are the “spokes” who provide the knowledge and guidance needed to provide SCD patients with proper care (Project ECHO, 2020).
Models Incorporating Community-Based Organizations
Another innovative model is the CDC-designed collaborative approach for addressing chronic care, which involves CBOs. This model uses four strategies: (1) epidemiology and surveillance, (2) environmental approaches that support health, (3) health system interventions to improve the use of clinical and preventive services, and (4) community resources linked to clinical services (Bauer et al., 2014). Employing similar models for SCD alongside an integration of behavioral and integrative health should be considered.
Financial and Socioeconomic Barriers
In a series of reports (IOM, 2001a, 2002a,b, 2003a,b, 2004), the IOM laid out the importance of health insurance to individual and community
health. It corrected the myths about the uninsured; delineated the effects of a lack of insurance on treatment for various serious conditions; emphasized the influence of the family on access to insurance and the effect of changes in family formation on insurance; delineated the effect of a lack of insurance on the health and well-being of individuals, families, and communities; and presented principles for reducing lack of coverage. Of particular importance to the current report, the reports emphasize the disproportionate burden on minority and low-income families and the changes in insurance coverage that can emerge with changes in employment or family composition, such the loss of family coverage for young adults. Although the reports do not examine SCD specifically, they do emphasize the importance of insurance in obtaining primary care and care for chronic illnesses. A later report found that there was an increase in the uninsured, especially low-wage earners, and an increase in restrictive policies for those with insurance (IOM, 2009). Thus, it would be expected that the type and content of health insurance would be a major influence on the ability of SCD patients to access medical care.
Private insurance is also variable. Until the Patient Protection and Affordable Care Act, private health insurance companies could use a preexisting condition as a basis for denying individual insurance coverage (KFF, 2019). Evidence from the CF population suggests that extending parental coverage for young adults to the age of 26 resulted in greater access to care, even though it does not automatically guarantee better health (Lanzkron et al., 2018b). Even with private insurance, coverage may vary due to changes in jobs or other reasons. Insurance may not be adequate for the individual’s needs, particularly durable medical equipment (NASEM, 2017), behavioral health care (IOM, 2003c), and dental care (Berdahl et al., 2016).
Finally, not all types of insurance may be considered equivalent. Anand et al. (2017) reported that among children undergoing hematopoietic stem cell transplant, those with private insurance were more likely to receive it. Previous sections of this chapter have also noted the variation in state Medicaid plans for both general services and CSHCN.
As suggested by the enabling services, other factors influence access to care, including specialist availability, transportation needs, the availability of comprehensible patient materials, and, potentially, the need for translation services. As noted in Chapter 2, a number of social factors influence access to care, including stigma, bias, and lack of public awareness.
SERVICES FOR SCT
Screening in the United States occurs in newborns, during pregnancy, and before participation in National Collegiate Athletic Association Division I, II, and III sports. Other opportunities for screening should occur for immigrants. The purpose of screening is two-fold: (1) to detect SCD and establish appropriate referrals for those with it and (2) to detect those at risk for having children with SCD.
SCT is associated with medical complications. Individuals with SCT have an increased risk for chronic renal disease, pulmonary embolism, and rhabdomyolysis (Naik et al., 2018). Recent data support the possibility of an association between SCT and atrial fibrillation (Douce et al., 2019).
Genetic counseling for families with children with SCT and for individuals with SCT improves the understanding of the risk of having offspring with SCD. In this context, rapid screening tests that use chemical reactions to determine the presence of HbS are not sufficient to provide accurate information for genetic counseling. The best test for hemoglobinopathies is a hemoglobin electrophoresis that quantifies the proportion of normal and variant hemoglobins. When accompanied by a complete blood count, better evaluations regarding the beta thalassemia trait are possible.
Screening for SCT and other hemoglobinopathy traits should be performed with genetic counseling before and during pregnancy. The American College of Obstetricians and Gynecologists (ACOG) recommends screening with a hemoglobin electrophoresis and complete blood count if there is a suspicion of hemoglobinopathy based on ethnic background (ACOG, 2017). A 2007 ACOG bulletin recommended that parents who are determined to be SCT carriers be provided genetic counseling to better inform their decisions regarding reproduction and prenatal genetic testing (ACOG, 2007). More research is needed on the use of genetic counseling for individuals with SCT (Pecker and Naik, 2018).
CONCLUSIONS AND RECOMMENDATIONS
There is a paucity of data concerning access to and the use of health care by children and adults with SCD. The impact of socioeconomic and psychosocial factors, including comorbid conditions and mental health condition needs, should be addressed in this population. The lack of such information will make it difficult to plan needed services and assess their impact. Thus, one part of a strategic plan would be to define and implement systems to gather the information related to health care use and the organizational health care infrastructure and the CBO infrastructure needed to provide high-quality care. One suggestion, as described in Chapter 3 and this chapter, would be to develop registry and surveillance systems
that collect information on not only patient demographics and metrics but also providers, services, and organizational infrastructure. Another might be to leverage existing national surveys to focus on SCD by combining years of data to obtain a sufficient sample. A third would be to exploit administrative datasets (e.g., insurance, Medicaid) to obtain a clearer picture of existing health care use among people with SCD (Grosse et al., 2010). Models of care should be explored to determine whether any seem superior for addressing SCD, given the barriers to care and the need to take into account health worker shortages (see Chapter 6). However, the benefit of pre-existing relationships among the SCD community, hospitals, and CBOs should be exploited. CDC’s strategic model for chronic illness delivery that incorporates community resources linked to clinical services should be explored further. These concepts must be explored in the context of increasing access to high-quality health care.
Conclusion 5-1: The available evidence suggests that the receipt of comprehensive care from a medical home is associated with fewer emergency department visits and hospitalizations for children with SCD. The receipt of comprehensive care involving a hematologist is also associated with greater receipt of preventive services for children with SCD. While children with SCD are cared for by a pediatric hematologist, they transition to adult health care providers who may or may not have experience with providing care for SCD.
Conclusion 5-2: There are no publications on the status of dental health among individuals with SCD. Considering that a substantial portion of the SCD population is covered by public insurance, it is important to note that the U.S. public insurance system does not universally provide dental coverage, so there are significant barriers to access to dental care for individuals with SCD.
Conclusion 5-3: By the time individuals with SCD reach adulthood, the majority (59 percent) of them have developed end-organ damage involving at least one organ, thus necessitating care delivery from a primary care and adult sickle cell specialist and other providers to ensure comprehensive SCD care delivery and to improve outcomes.
Conclusion 5-4: Individuals with pain are more likely to manage their pain at home for a variety of reasons. However, home opioid use is variable and related to having more pain, more crises, and higher acute care use, thus offering an opportunity for research to help individuals with SCD establish structured regimens for managing care at home.
Conclusion 5-5: The transition from pediatric to adult care is a critical time for individuals with SCD. Young adults experience increased disease complications and end-organ damage, face loss of health insurance and of the usual source of care, and feel inadequately prepared to manage their disease. There are models of transition for other diseases that can inform coordinated transitions for individuals with SCD.
Conclusion 5-6: Mental health care is significantly compromised in SCD partly due to the chronic burden of disease and the impact of pain and pain treatments on the brain. There is a lack of good evidence regarding the natural history of mental health in SCD and detailed information on the psychological and psychosocial effect on patients as they age into adults.
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