Allogeneic hematopoietic stem cell transplant for sickle cell disease: The why, who, and what.

ABSTRACT

Allogeneic hematopoietic stem cell transplants (allo-HSCTs) from matched-related donors (MRDs), mismatched-related donors (MMRDs), and matched-unrelated donors (MUDs) are increasingly being used to treat sickle cell disease (SCD) in both pediatric and adult patients. The overall results have been extremely encouraging, especially if a MRD is available and the transplant being performed before the age of 13. Although there is a general consensus that patients with high-risk SCD, even in adults and irrespective of donor characteristics, should be offered allo-HSCT, the debates on optimal patient selection and timing of transplant have yet to be resolved. Unlike patients with hematologic malignancies, there are also a number of clinical issues that require to be addressed in patients with SCD undergoing allo-HSCT. In this review, we will discuss the reasons allo-HSCT should be offered more widely to patients with SCD, the challenges facing physicians in patient selection and timing of transplant, and the awareness of and solutions to prevent the complications that are unique or more common in SCD undergoing allo-HSCT.