Chayamon TakpraditSusan E. ProckopNancy A. KernanAndromachi ScaradavouKevin CurranJulianne RuggieroNicole ZakakRichard J. O'ReillyFarid BouladNew York Presbyterian HospitalFaculty of Medicine, Siriraj Hospital, Mahidol UniversityMemorial Sloan-Kettering Cancer Center2019-08-282019-08-282018-01-01Journal of Pediatric Hematology/Oncology. Vol.40, No.4 (2018), e220-e22415363678107741142-s2.0-85046247549https://repository.li.mahidol.ac.th/handle/123456789/47199© 2018 Wolters Kluwer Health, Inc. All rights reserved. Background: The combination of cyclophosphamide (CY) and antithymocyte globulin (ATG) has been used as a standard conditioning regimen for matched related donor transplantation in patients with severe aplastic anemia. Procedure: To decrease the regimen-related toxicity while maintaining appropriate engraftment and survival rates, fludarabine (FLU) was added to the regimen. Four pediatric patients received matched related donor bone marrow transplantation with CY (50 mg/kg×2) (instead of the 50 mg/kg×4 standard dosing), equine ATG (30 mg/kg×3), with the addition of FLU (30 mg/m 2 ×4). Graft versus host disease (GvHD) prophylaxis included a calcineurin inhibitor and methotrexate. Results: No grade 4 acute toxicities occurred during the first 30 days after transplant. All patients engrafted with normalization of peripheral blood counts and transfusion independence. One patient developed grade 1 to 2 acute GvHD, followed by chronic GvHD that resolved. With a median follow-up of 41.7 months, all 4 patients are alive and transfusion free, with complete donor chimerism. This combination of a low-dose CY/ATG+FLU regimen was overall very well tolerated and contributed toward a successful outcome including engraftment, chimerism, and survival. Conclusion: This small pilot study shows that this cytoreductive regimen could be considered as the standard of care for transplantation of pediatric patients with aplastic anemia from HLA-matched siblings.Mahidol UniversityMedicineArticleSCOPUS10.1097/MPH.0000000000001106