Stem cell transplantation for sickle cell disease: Can we reduce the toxicity?

Since the genetic basis of sick le cell anemia was discovered over 50 years ago, many therapies have Since been developed for the treatment of this di sorder. Hematopoietic cell transplantation offers curative potential, but i t is associated with a 5-10% risk of dying. Patients who undergo allografti ng but develop stable donor-host hematopoietic chimerism appear to experien ce a significant clinical benefit. Our paper discusses the risks and benefi ts of hematopoietic cell transplantation in patients with sickle cell disea se and summarizes the outcome of 147 patients who received allografts for s ickle cell disease. We also review the development of new approaches to est ablish stable mixed chimerism after transplantation for sickle cell disease .