Exploring the State of the Science of Stem Cell Transplantation and Posttransplant Disability Proceedings of a Workshop (2022) / Chapter Skim
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1 Introduction
1 Introduction
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. In the opening remarks, Rosenbaum explained that the purpose of the workshop was to facilitate a discussion to examine disability following hematopoietic stem cell transplantation (HSCT)
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; • Supplemental treatments used to improve a person's phy sical or mental functioning following stem cell treatment or transplantation, and the settings in which the treatments are provided; • The typical length of time from the initiation of stem cell treat ment or transplant surgery until the person's functioning im proves to the point of which the condition is no longer disab ling, and specific ages or other recipient traits where impro vement is more likely; • Laboratory or other findings used to assess medical and func tional improvement after stem cell treatment or transplantation; and • Recent medical advances, promising research, or new technologies that may alter expected patient outcomes, and potential advances anticipated in the near future.
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Finally, the fourth session looked forward at the future outlook for HSCT and related disability. This Proceedings of a Workshop describes the presentations from invited subject-matter experts and panel discussions held during the workshop, including responses to questions from the SSA and the general public.
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• Patient care includes multidisciplinary care teams and services that help to support recovery and mitigate impairment from HSCT, such as prehabilitation (to improve patients' strength before a transplant) , rehabilitation, physical therapy, mental health care, home care, social support, employment assistance, and palliative care.
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• Patients receiving autologous transplants recover more quickly and have fewer late effects, but their self-reported health at 1 year is similar to patients who received allogeneic transplants.
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• Several diagnoses are associated with high burden of late disability after HSCT in children, including: leukodystrophies, mucopolysacc haridoses, sickle cell anemia, Fanconi anemia, dyskeratosis con genital, and brain tumors.2 (Davies) • Children who receive a transplant have heightened health risks that can continue throughout life, including a risk of chronic conditions (ranging from hypertension to severe physical limitations)
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Emerging Treatments and Research to Improve HSCT Success • CAR T-cell therapy, which targets cancer cell proteins to kill the cell, is curing previously incurable, rapidly progressing, life-threa tening malignancies, such as relapsed or refractory acute lymp hocytic leukemia. There are now five Food and Drug Administration (FDA)
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• HSCT is the one proven therapy for curing sickle cell disease; now an emerging approach is to edit the patient's cells in order to correct the genetic mutation. This gene therapy approach in patients with sickle cell disease, if successful, results in normal hemoglobin production from the cells that were infused.
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, they share the same medical criteria and use the same sequential evaluation processes to determine initial eligibility. For adults, said Nibali, disability is defined by statute as the "inability to do any substantial gainful activity by reason of any medically determinable physical or mental impairment which can be expected to result in death or last for a continuous period of not less than 12 months" (C.F.R.
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In response to a question from a participant, Nibali noted that SSA has medical consultants to provide advice on CDR decisions, but he does not know how many of these consultants have a background in stem cell treatment. The discussions at this workshop, said Nibali, will assist SSA in improving and refining its evaluation processes, as well as contribute to the agency's mission to provide the most accurate disability decisions as efficiently as possible.
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