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Transplantation for Sickle Cell Disease
Sponsoring Organization: The American Society of Hematology (ASH)
Background and Objective
Sickle cell disease can have devastating clinical consequences, from anemia, vaso-occlusion, end organ insult, and thrombosis. The therapeutic landscape is finally extending beyond hydroxyurea, which has important clinical benefits. Hematologists now have access to L-glutamine, crizanlizumab, and voxelotor, which help ameliorate pain and improve anemia. However, these agents do not lead to cure. Hematopoietic stem cell transplantation (HSCT) can be curative and is a consideration for selected patients. A panel convened by ASH developed recommendations for HSCT in the clinical care of patients with sickle cell anemia; recommendations were considered strong if the term “recommends” was used or conditional if the term “suggests” was used.
Key Points
- HSCT is suggested for patients who have had overt stroke or abnormal transcranial Doppler examinations.
- HSCT is suggested for patients with frequent pain and an inadequate response to standard therapies.
- HSCT is suggested for patients with recurrent acute chest syndrome.
- In eligible patients, use of HSCT is suggested at a younger rather than older age.
- For patients without a matched sibling donor, transplant from an alternative donor is suggested.
Comment
Given the high risk yet high reward associated with transplantation in sickle cell disease, guidance is welcomed. These guidelines will help hematologists identify potential candidates, particularly those with overt stroke, recurrent acute chest syndrome, and uncontrolled pain despite standard treatment. The guidelines also provide some direction for transplant physicians regarding the ideal donor and conditioning regimens. However, given the available evidence, the recommendations are conditional rather than strong. Timing of transplantation remains a challenge — younger patients fare better than older patients, who may have limiting comorbidities such as chronic kidney disease and pulmonary hypertension. While there are new therapies that may benefit quality of life, and emerging therapies (e.g., gene therapy) that may alter the natural history of the disease, HSCT still should be considered for selected, eligible candidates. These guidelines add more clarity to the decision-making process.
Citation(s)
Author:
Kanter J et al.
Title:
American Society of Hematology 2021 guidelines for sickle cell disease: Stem cell transplantation.
Source:
Blood Adv
2021
Sep
28; [e-pub].
(Abstract/FREE Full Text)
Empfohlen von
Brady L. Stein, MD, MHS