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1 Introduction
Pages 19-46

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From page 19...
... , necessitating action to improve health and outcomes and reduce this disparity. 1  This text has changed since the prepublication release of this report to more accurately reflect the estimates of prevalence of sickle cell disease identified in the literature.
From page 20...
... The Committee on Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action was established in response to this request. As part of its work, the committee was asked to develop a framework that provides guidance on the best approaches to addressing pertinent issues in SCD such as health care disparities, stigma, race and biases, access to care, workforce development, transitions in care, innovations needed, curative treatments, and the role of patient advocacy and community engagement.
From page 21...
... The committee will provide guidance on priorities for programs, poli cies, and research and make recommendations, as appropriate, regarding: • limitations and opportunities for developing national SCD patient registries and/or surveillance systems; • barriers in the health care sector associated with SCD and SCT, in cluding access to care and quality of care, workforce development, pain management, and transitions from pediatric to adult care; • needed innovations in research, particularly for curative treatments such as gene replacement/gene editing and increasing awareness and enrollment of SCD patients in clinical trials; and • the expanded and optimal role of patient advocacy and commu nity engagement groups. Committee guidance should be formulated around strategic objec tives (strategic plan)
From page 22...
... Conceptual Approaches for Information Gathering Life-Span Approach SCD causes various challenges at different stages in life, so the committee conceptualized the tasks for this report by assessing the needs of people living with SCD over the life span. For example, dactylitis occurs mainly in children, whereas heart failure and chronic leg ulcers typically affect adults.
From page 23...
... Person-Centric SCD Care Approach Considering that more people with SCD are living into adulthood, it is important to understand how to manage this chronic disease across the life span, with collaborative input from physicians, those living with SCD and their families, and relevant community stakeholders. The committee decided that effective management of SCD needs to happen in the context of a team-based comprehensive care model that places the patient and his or her needs at the center.
From page 24...
... SCD = care cell disease. SOURCES: IOM, 2012; originally adapted from Wagner, 1998.
From page 25...
... . Similar care models for a variety of chronic diseases have been implemented, such as for cystic fibrosis (CF)
From page 26...
... . If both parents have SCT, each child will have a 50 percent chance of inheriting SCT and a 25 percent chance of inheriting SCD, while there is a 25 percent chance that the child will inherit neither SCT nor SCD and have non-mutated hemoglobin.
From page 27...
... cell disease) contributed to the spread of the disease across the world (Dampier, 2019; Piel et al., 2010; Schroeder et al., 1990; Solovieff et al., 2011; Williams and Weatherall, 2012)
From page 28...
... L Hassell, Population Estimates of Sickle Cell Disease in the U.S., S512–S521, Copyright (2010)
From page 29...
... . Despite medical advances, however, individuals living with SCD continue to experience barriers to access care and knowledgeable providers, and their average life expectancy remains 20–30 years lower than that of the average American (Lubeck et al., 2019; Piel et al., 2017)
From page 30...
... . Early death occurs in all SCD genotypes, including the compound heterozygous sickle cell syndromes, particularly during the delicate transition period from pediatric to adult care (Blinder et al., 2013)
From page 31...
... SOURCE: Global Health Data Exchange, 2019. 29,284 years of life lost, and 3,984 disability-adjusted life-years (DALYs)
From page 32...
... . In developing countries, the burden of SCD is high; 90 percent of children do not live into adulthood (Sickle Cell Disease Coalition, n.d.)
From page 33...
... . Finally, health care personnel issues and resources play a role, as SCDexpert-led, multidisciplinary health care teams focusing on comprehensive care appear to be more prevalent and accessible in pediatrics than in adult care (Treadwell et al., 2011)
From page 34...
... to conduct a demonstration program to develop and establish systemic mechanisms, including a national coordinating center, to improve the prevention and treatment of SCD. This act established the Sickle Cell Disease Treatment Demonstration Program, with the purpose of funding regional coordinating centers that
From page 35...
... 1972 2002 2004 2013 2018 National Sickle Cell Anemia The American Jobs Creation Act of The Sickle Cell Disease & Other Control Act of 1972 2004 Heritable Blood Disorders (Public law no.
From page 36...
... The most recent legislative action for SCD took place on December 18, 2018, with the signing into law of the Sickle Cell Disease and Other H ­ eritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018 (Public Law 115-327)
From page 37...
... NOTE: FDA = U.S. Food and Drug Administration; SCD = sickle cell disease.
From page 38...
... NOTE: CF = cystic fibrosis; SCD = sickle cell disease. SOURCE: Adapted from Farooq and Strouse, 2018.
From page 39...
... ;  •  he Health Resources and Services Administration T currently funds programs for children with inheritable disorders, such as SCD, including the Sickle Cell Disease Treatment Demonstration Program and the Newborn Screening Follow-up Program (see Chapter 3) ;  •  he National Institutes of Health, through the National T Heart, Lung, and Blood Institute, conducts clinical trials and research and also funds extramural research on SCD and launched the Cure Sickle Cell Initiative in 2018 with the goal of accelerating the development of genetic therapies for SCD;  •  he U.S.
From page 40...
... The roles of these organizations also vary and are discussed in Chapter 8. Patients and Families Individuals living with SCD and their families are integral to improving SCD care because of their lived experience with the disease.
From page 41...
... This chapter delves into how and where people with SCD should receive care and what types of care they should receive; the issues with the transition from pediatric to adult care; what comprehensive SCD care encompasses; and the geographic, financial, and socioeconomic barriers to care. Chapter 6 addresses the current state of quality of SCD care and the workforce needs to deliver high-quality care.
From page 42...
... 2015. 47 causes of death in adult sickle cell disease patients at Howard University.
From page 43...
... 2019. Estimated life expectancy and income of patients with sickle cell disease compared with those without sickle cell disease.
From page 44...
... 2016. Defining sickle cell disease mortality using a population-based surveillance system, 2004 through 2008.
From page 45...
... 2011. Ancestry of African Americans with sickle cell disease.


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