The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.
Patients must be ≥ 12 months and < 19 years of age at the time of study enrollment.
homozygous Hb S disease (HbSS),
sickle-Hb C disease (HbSC),
sickle beta-plus-thalassemia (HbS/β+), or
Patients who are unable to comply with or follow the study protocol.
Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.