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

Background&Objectives:Hematopoietic stem cell transplantation (HSCT) offers potentially curative treatmnt for bone marrow failure syndromes (BMFS). We aimed to evaluate the BMFS-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. Methods: This study retrospectively analyzed 147 patients who underwent stem cell transplantation for BMFS (congenital or acquired) in Nasser institute, between January 2009 and December 2015.Results: Patients{u2019} age at the time of transplantation ranged from 1.0 to 18.0 years; with a mean of10.5±4.9 and median 10 years. Ninety four were males (M/F ratio: 1.7). Out of the 147 patients; 10 (6.8%) patients received bone marrow and 137(93.2%) received peripheral blood stem cells (PBSC) harvest. All donors were HLA identical with 137 matched sibling donors and 10 matched parent donors. Transplant related mortality was 23.8% and the most common cause of death was infection in 57.1% of cases.The probabilities of 5- year overall survival (OS) was 73.4%,disease free survival (DFS) was 74.2 % andevent free survival (EFS) was 70.4%. OS and DFS were significantly worse in patients who had aGVHD (p=0.001, 0.001 respectively)

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