Hematopoietic stem-cell transplantation for sickle cell disease: current evidence and opinions.

With rapidly expanding evidence of benefit reported by several groups, allogeneic hematopoietic stem-cell transplantation has become an acceptable treatment option for sickle cell disease. It is currently the only curative therapy available. Hematopoietic stem-cell transplantation was offered primarily as a therapeutic option for severe sickle cell disease in the context of myeloablative matched sibling donor transplants over the last two decades and helped to establish the benefits of transplantation for this disorder. While this approach provided proof of principle, the disadvantages and limitations of transplantation became evident along the way. It has been recognized that transplantation for sickle cell disease does not need to adhere strictly to the principles of transplantation for malignant disorders, such as achievement of full donor cell chimerism. As reviewed here, in recent years the transplant community has set out to explore ways to make stem-cell transplantation more available to patients with the disease, define indications and better timing, and offset toxicities with novel approaches to conditioning and better supportive care.