Long-Term Free Survival of Two Class III β-Thalassemic Patients after Non-Myeloablative Stem Cell Transplantation

Abstract

At present, hematopoietic stem cell transplantation is the only curative treatment for β thalassemic patients. Conventional myeloablative stem cell transplantation is associated with significant morbidity and mortality, and non-myeloablative stem cell transplantation is associated with high graft failure rate. Some modification in this treatment approach can result in successful transplantation in thalassemic patients. Two successful Fludarabine - based non-myeloablative stem cell transplantation in two Class III β thalassemic patients are reported here. The first patient was a 14-year old girl that developed rapid engraftment and full Chimerism after rapid tapering of cyclosporine as graft-versus-host disease (GVHD) prophylaxis drug according to our protocol. Another patient was a 24-year old female patient that developed cyclosporine toxicity, and early tapering of cyclosporine helped for rapid engraftment and successful transplantation. After these two successful experiments in non-myeloablative peripheral blood stem cell transplantation for our class III β thalassemic patients, we concluded that Fludarabine-based nonmyeloablative stem cell transplantation with adequate number of stem cells at the time of transplantation and rapid tapering of GVHD prophylaxis drugs after transplantation can potentially help for rapid engraftment and successful stem cell transplantation in high risk β-thalassemic patients.

Keywords

• β-thalassemia; Stem cell, Transplantation, Non-myeloablative, Fludarabine