Allogeneic hematopoietic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemia--a single-center analysis of 100 patients.

Abstract	The authors compared the outcome in 100 children (61 boys, 39 girls; median age of 10.1 +/- 3.4 years) with aplastic anemia who underwent either immunosuppressive therapy (IST; n = 70) or hematopoietic stem cell transplantation (HSCT; n = 30) between 1998 and 2007. Conditioning regimes for HSCT were a combination of either cyclophosphamide (Cy) with antilymphocyte globulin (ALG) or fludarabine (Flu) with Cy or busulfan (Bu) +/- antithymocyte globulin (ATG). Stem cell source was bone marrow in 20 and peripheral blood stem cells (PBSCs) in 10. Patients undergoing IST received either equine ALG or ATG in combination with steroids and cyclosporine. Primary engraftment was seen in 25 children (83.3%), with acute graft-versus-host disease (aGvHD) in 5 (16.6%). The day 100 transplant-related mortality (TRM) was 30% and at a median follow up of 36 months (range: 6-197), the overall and disease-free survival is 70%. Among children who received IST, 60 children received ALG while 10 received ATGAM. Responses were seen in 27 children (43.5%), which was complete (CR) in 12 and partial (PR) in 15. At a median follow up of 38 months (range: 1-84), the overall survival is 37.1%, with 81.4% survival among responders and <10% survival among non-responders. HSCT would be the treatment of choice in children with severe aplastic anemia who have a human leukocyte antigen (HLA)-matched related donor and is superior to IST in this series from India.
Authors	Biju George, Vikram Mathews, Auro Viswabandya, Kavitha M Lakshmi, Alok Srivastava, Mammen Chandy
Journal	Pediatric hematology and oncology (Pediatr Hematol Oncol) Vol. 27 Issue 2 Pg. 122-31 (Mar 2010) ISSN: 1521-0669 [Electronic] England
PMID	20201693 (Publication Type: Journal Article)
Chemical References	Immunosuppressive Agents
Topics	Anemia, Aplastic (diagnosis, drug therapy, surgery, therapy) Child Disease-Free Survival Female Hematopoietic Stem Cell Transplantation Humans Immunosuppressive Agents (therapeutic use) India Male Survival Rate Transplantation, Homologous

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