Exploring the State of the Science of Stem Cell Transplantation and Posttransplant Disability Proceedings of a Workshop (2022) / Chapter Skim
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2 Overview of Hematopoietic Stem Cell Transplantation and Disability in the United States
2 Overview of Hematopoietic Stem Cell Transplantation and Disability in the United States
Pages 11-24
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From page 11... ...
These cells are "multipotent" because they can form different types of cells within the tissue type; for example, skin stem cells can form hair, epidermis, or sweat glands. Hematopoietic stem cells -- the topic of this workshop -- are cells responsible for forming all blood and immune cells in the body.
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From page 12... ...
There are two major types of HSCT, said Horowitz. Autologous transplants are collected from the patient's own bone marrow or bloodstream; this is an option when dose-intensification is the primary goal.1 Allogeneic transplants use cells from a family member, unrelated donor, or umbilical cord blood; this type of transplant has the potential for potent immune-mediated TABLE 2-1 Diseases Treatable with Hematopoietic Stem Cell Transplantation Malignant Disease Nonmalignant Disease • Leukemias and lymphomas • Severe aplastic anemia and other marrow • Myelodysplastic syndromes failure states • Multiple myeloma and other • Inherited immune disorders (e.g., severe plasma cell disorders combined immunodeficiency)
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From page 13... ...
The risk of recurrence or FIGURE 2-1 Annual number of hematopoietic stem cell transplant recipients in the United States by transplant type. NOTE: HCT = hematopoietic cell transplantation.
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FIGURE 2-2 Relapse or progression after allogeneic hematopoietic stem cell transplantation for hematologic malignancies in U.S. patients under 60 years, 2015–2019.
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From page 15... ...
In conclusion,Horowitz said that HSCT is an effective therapy for patients with a wide range of hematologicdisorders, but rates of mortality and morbidity following a transplant are stillsubstantial. PATTERNS OF RECOVERY Following Horowitz's presentation, Stephanie Lee, a member of the FredHutchison Cancer Research Center, gave workshop participants an overview of the patterns ofrecovery seen after patients are treated with HSCT.
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From page 16... ...
Long-term effects are issues that begin during the transplant and persist, while late effects are issues that arise after the transplant but are attributable to it. In the first year after HSCT, said Lee, three main factors determine a FIGURE 2-4 Attributable disability after hematopoietic stem cell transplantation.
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From page 17... ...
One older study (Lee et al., 2001) asked patients to rate their agreement with the statement "I have recovered from my transplant." Patients whohad undergone autologous transplants were much more likely than patients receivingallogeneic transplants to report they had recovered by 6 months, but by 24 months, the populationsreported similar recovery rates of around 70 percent (Figure 2-7)
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From page 18... ...
18 STEM CELL TRANSPLANTATION AND POSTTRANSPLANT DISABILITY FIGURE 2-5 Physical and mental functioning following hematopoietic stem cell transplantation. SOURCES: As presented by Stephanie Lee, November 15, 2021; Jim et al., 2016.
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From page 19... ...
In addition to fatigue, Lee saidthat transplant survivors face a wide variety of late effects and comorbiditiesincluding neuropsychological effects, cancer, infection, and FIGURE 2-7 Self-reported recovery outcomes following hematopoietic stem cell transplantation. NOTE: auto = autologous transplant; allo = allogeneic transplants SOURCES: As presented by Stephanie Lee, November 15, 2021; Lee et al., 2001.
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From page 20... ...
There are myriad causes of disability among transplant survivors, said Lee, and thesedisabilities can prevent or delay a return to work. Theseinclude: • Physical dysfunction, such as lack of strength and stamina FIGURE 2-8 Potential comorbidities and late effects following hematopoietic stem cell transplantation.
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From page 21... ...
74% @ 3 years; older autos with lower pre-HCT income, Wong et al., 2020 allos with chronic GVHD Longitudinal N = 690, 79% auto 62% @ 1 year; hospitalizations, relapse, pain, fatigue Morrison et al., 2016 Longitudinal N = 152, allo 47% @ 5 years; peripheral blood Lee et al., 2016 Cross-sectional N = 203, > 5 year, 85% allo 77% @ 12 years; older, living alone, physical/mental late Tichelli et al., 2017 survey effects, fatigue Cross-sectional N = 1,048, ages 20–64, > 2 52–76% @ 5 years; female, older, part-time Kurosawa et al., 2021 survey year allo DFS CIBMTR study N = 2,844 childhood HCT, 73% when 28–32 years old; RIC/NMA, older at HCT Bhatt et al., 2019 1985–2010, allo CIBMTR study N = 1,365 young adults, 50% @ 1–3 years; female, multiple comorbidities, severe Bhatt et al., 2021 2008–2015, allo acute GVHD, relapse NOTE: auto = autologous transplant; allo = allogeneic transplants; DFS = disease-free survival; GVHD = graft-versus-host disease; HCT = hematopoietic stem cell transplantation; RIC/NMA = reduced-intensity conditioning/nonmyeloablative (i.e., therapies given in conjunction with HSCT)
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From page 22... ...
• Patients receiving autologous transplants recover more quickly and have fewerlate effects, but their self-reported health at 1 year is similar to patients whoreceived allogeneictransplants. • Transplant patients have a high burden of comorbidities, ongoing medical care,and compromised function and quality of life.
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From page 23... ...
Appelbaum said that the data that Lee presented on the similarity in outcomesbetween patients receiving autologous and allogeneic transplants were surprising; he said he wouldhave expected patients undergoing autologous transplants to have better outcomes. Lee said there isa bias in the field that expects better outcomes from autologous transplants, but that the data donot reflect this.
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From page 24... ...
Q5: What would you tell a patient if he or she asked, "Why should I do a hematopoietic stem cell transplant? " For many patients, said Appelbaum, transplantation is the only opportunity to cure.For example, patients with high-risk acute myeloid leukemia have "no chance for cure" oncethey have relapsed, other than HSCT.
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