Thesis (M.Sc.) - Cairo University - Faculty of Medicine - Department of Pediatrics

Hematopoietic stem cell transplantation (HSCT) offers curative potential for Ý-thalassemia major. To evaluate the thalassemia-free survival and outcome of stem cell transplantation in the Egyptian experience and to analyze the patients and donors{u2019} characteristics as well as transplantation related factors that could affect the graft success rate. This study retrospectively analyzed 174 patients who underwent stem cell transplantation for Ý-thalassemia major in Nasser institute, between January 1997 and December 2014. Their age at the time of transplantation ranged from 0.7 to 23.7 years; with a mean of 6.1±4.2 and median 4.6 years. One hundred and six were males (60.9%) and 68 (39.1%) females (M/F ratio: 1.6). Out of the 174 patients; 38 (21.8%) were in class I, 110 (63.2%) were in class II, and 26 (14.9%) were in class III. Twenty-six (14.9%) patients received bone marrow and 148 (85.1%) received peripheral blood stem cells (PBSC) harvest. The donors were selected based on degree of human leukocyte antigen (HLA) matching, and all of them were HLA-identical donors. 153 were matched sibling donors (MSD) and 10 were matched parent donors (MPD). The study showed that the probabilities of 5-year overall survival (OS) is 74.9%, disease free survival (DFS) is 61% and transplant related mortality (TRM) is 18.4%. Graft failure occurred in 12.6%, and the use of PBSC showing lower incidence of graft failure (GF) than BM harvest. The optimum age for transplantation is from 2-6 years, PBSC harvest and higher stem doses have more favorable outcomes and stem cell source was the main risk factor of graft failure

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