Hematopoietic stem cell transplantation (HSCT) is currently the only established cure for sickle cell disease (SCD). Replacement of the stem cell that has the defective beta globin allele with the normal gene decreases hemoglobin S and the risk of complications of SCD. The first case reported was a girl with acute myeloid leukemia and SCD who received HSCT and achieved long-term SCD and leukemia-free survival. Given the favorable outcomes of HSCT with thalassemia major using myeloablative preparative regimens, this approach became widely used in the initial studies of HSCT in SCD. The current standard of care is to use a myeloablative stem cell transplantation in patients with severe disease who have human leukocyte antigen–identical sibling. HSCT improves organ function, quality of life, and overall and disease-free survival. However, this is associated with high risk of gonadal dysfunction and graft versus host disease in addition to the mortality associated with the myeloablative HSCT. Reduced-intensity HSCT has also been reported with high rates of engraftment and favorable outcomes. This has been introduced to lower the gonadal dysfunction, mortality, and graft versus host disease associated with myeloablative approaches. Other approaches include HSCT using matched unrelated donors, cord blood units, and human leukocyte antigen haploidentical donors. Unfortunately, graft rejection is a common complication with these approaches. In this chapter, we review the indications of HSCT for SCD and outcomes of different transplant strategies including alternative donor transplant, graft rejection, and infertility after transplantation.
Part of the book: Sickle Cell Disease