Outcome and late effects of matched related peripheral-blood stem-cell transplantation in children with severe aplastic anemia

Abstract

Background Aplastic Anemia (AA) is a life- threatening disease. Cure can be achieved in a large proportion of patients with prompt treatment with matched sibling donor Hematopoietic Stem Cell Transplantation (HSCT) as first line therapy. Aim Study of the outcomes and late effects among children with severe aplastic anemia after receiving matched related peripheral-blood stem-cell (PBSC) transplantation and cyclophosphamide/antithymocyte globulin conditioning regimen at Ain Shams University Pediatric Bone Marrow Transplantation Unit, Cairo, Egypt. Methods Data of 15 patients with severe aplastic anemia who received matched related PBSC transplantation between January 2011 and December 2018 were analysed 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 the liver, kidney, pulmonary, cardiac, endocrine functions, chronic graft-versus-host disease (GVHD), 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 posttransplant. Blood counts were within normal limits. Chimerism was complete for all except one. Growth assessment revealed Z score of weight, height, and BMI 0 (−1.8–1.6), −0.7 (−1.8–0.95), and 0.34 (−1.29–2). Abnormal thyroid function was found in three (27%) patients. No documented high-grade acute GVHD or extensive chronic GVHD. 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 PBSC transplantation in the pediatric population in low-income countries. Overall survival and event-free survival were satisfactory.