الفهرس | Only 14 pages are availabe for public view |
Abstract ABSTRACT Background: Aplastic anemia (AA) is life threatening disorder in pediatric age group with an increasing incidence nowadays. Hematopoietic stem cell transplant being the 1st line therapy. We aimed to study the outcomes and late effects among children with severe aplastic anemia after receiving matched related peripheral blood stem cells transplantation and Cyclophosphamide / Antithymocyte globulin conditioning at Ain Shams University Pediatric bone marrow transplantation Unit, Cairo, Egypt. Methods: Data of 15 patients with severe aplastic anemia received matched related peripheral blood stem cells transplantation between January 2011 and December 2018 were analyzed retrospectively. All long- term survivors were followed up for 2 years. Follow-up studies included detailed assessment of growth, graft evaluation, immune reconstitution, long term effects on liver, kidney, pulmonary, cardiac, endocrine functions, chronic graft-versus-host disease and infectious complications. Results: The median duration of the study was 87 months (43-115 months). The overall survival was 73.3%. Event free survival was 66.7%. All mortality was within 2 months post-transplant. Blood counts were within normal limits. Chimerism was complete for all except one. Growth assessment revealed Z score of weight, height, and body mass index 0(-1.8-1.6), -0.7 (-1.8-0.95) and 0.34 (-1.29-2). Abnormal thyroid function was found in 3 patients (27%). No documented high grade acute graft-versus-host disease or extensive chronic graft-versus-host disease. Cardiac, Pulmonary, Hepatic and Renal functions showed no abnormality. Immune reconstitution was consistent, and no malignant complications were reported. Conclusion: This study confirmed the feasibility, and safety of matched related peripheral blood stem cells transplantation in the pediatric population in low-income countries. Overall survival and event free survival were satisfactory |