Peripheral Blood Stem Cell Transplant For Homozygous f3- Thalassemia : Evaluation Of Engraftment

Abstract

Homozygous -thalassemia is a widely distributed disease causing a significant incidence of morbidity and mortality. Ever since the early success reported in the 1980s for BMT as a treatment method, there has been increasing interest to improve and update the services offered to the thalassemic child. Our study conducted at "Nasser Institute of Research and Therapy" included 18 thalassemic children who underwent peripheral stem cell transplants and 2 patients who received a conventional BMT. All our patients received busulfan, cyclophosphamide and anti- thymocyte globulin for conditioning. Cyclosporine A and a short course of methotrexate were given as anti-GVHD prophylaxis. Engraft:ment was precisely evaluated using the VNTR technique. Sixteen patients engrafted while 4 patients experienced 1ry or 2ry graft failure. GVHD and VOD were each diagnosed in 2 patients. The overall and disease free survival are 95% and 80% respectively at a mean follow up period of2 ± 1 years (range 6mths to 4.5