HCT using an innovative conditioning regimen can result in a cure with marked improvements in quality of life for patients with high-risk sickle cell disease, according to a study of 13 adults transplanted using matched related donors. Patients were conditioned with alemtuzumab and total body irradiation, with sirolimus for post-HCT immunosuppression. At a median follow-up of 22 months (range, 12 to 44) no patients experienced acute or chronic GVHD, and all patients were alive. At 1 year post-transplant, 12 of 13 patients had stable donor chimerism, and these patients had normalized hemoglobin concentrations, with improved cardiopulmonary and quality of life parameters, including bodily pain, general health, and vitality.
Saraf SL, et al. Biol Blood Marrow Transplant