Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that causes significant complications and affects the quality of life. Hematopoietic stem cell transplantation (HSCT) is the sole curative intervention for SCD; however, there is a lack of guidelines to inform its application in clinical practice.
Recent evidence-based guidelines of the American Society of Hematology (ASH) support patients, clinicians, and health professionals in deciding on HSCT for SCD. It issued eight recommendations, with low or very low certainty evidence due to the lack of RCTs for HSCT in SCD, the scarcity of universal endpoints used in HSCT trials, and the shortage of direct comparative therapies.
The panel recommends using MSD HSCT for all individuals at risk of neurologic injury, recurrent vasoocclusive pain crises, or a history of recurrent ACS. Furthermore, when feasible, HSCT should be completed at the earliest age possible.
The benefits and burdens of HSCT direct that all patients (even those without an MSD) with severe complications of SCD should receive information about transplantation as an option. It recommends undergoing HSCT with an MSD and nonmyeloablative therapy for adults with SCD.
Overall, alternative donor HSCT and newer nonmyeloablative regimens should be undertaken for clinical trials to better evaluate the efficacy and outcomes for future recommendations. Future research should also include the development of a robust SCD registry to serve as a comparator for HSCT studies.
Kanter J, Liem RI, Bernaudin F, Bolaños-Meade J, Fitzhugh CD, Hankins JS, Murad MH, Panepinto JA, Rondelli D, Shenoy S, Wagner J, Walters MC, Woolford T, Meerpohl JJ, Tisdale J. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation. Blood Adv. 2021 Sep 28;5(18):3668-3689. DOI: 10.1182/bloodadvances.2021004394C.
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