New evidence-based guidelines to help patients and providers understand how timing and type of hematopoietic stem cell transplantation (HSCT) plays a role in managing individuals with sickle cell disease (SCD), according to a press release published online by the American Society of Hematology on September 28, 2021.
The new guideline contains eight recommendations formulated using the GRADE Evidence-to-Decision frameworks.
“The evidence for all recommendations is of low or very low certainty because of the lack of randomized control trials for HSCT in SCD, the lack of universal endpoints used in HSCT trials, and the lack of direct comparative therapies,” the report says.
The report further states that HSCT with matched sibling donor (MSD) be considered for all individuals at risk of neurologic injury or those in frequent pain or history of recurrent acute chest syndrome. Furthermore, the panel recommended that HSCT with MSD and non-myeloablative therapy should be used for adults with SCD.
The panel also agreed that when possible, HSCT should be done at an earlier age because aging is associated with an increased risk of irreversible SCD-related damage to the body in patients who require transplantation. The panel held a stronger view that all patients with severe complications of SCD should be given the option of transplantation.
The panel concluded that alternative donor HSCT and newer non-myeloablative regimens need clinical trials to demonstrate their efficacy and outcomes before future recommendations can be made.
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