Publication: Allogeneic Matched Related Donor Bone Marrow Transplantation for Pediatric Patients with Severe Aplastic Anemia Using "low-dose" Cyclophosphamide, ATG Plus Fludarabine
Issued Date
2018-01-01
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ISSN
15363678
10774114
10774114
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2-s2.0-85046247549
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Mahidol University
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SCOPUS
Bibliographic Citation
Journal of Pediatric Hematology/Oncology. Vol.40, No.4 (2018), e220-e224
Suggested Citation
Chayamon Takpradit, Susan E. Prockop, Nancy A. Kernan, Andromachi Scaradavou, Kevin Curran, Julianne Ruggiero, Nicole Zakak, Richard J. O'Reilly, Farid Boulad Allogeneic Matched Related Donor Bone Marrow Transplantation for Pediatric Patients with Severe Aplastic Anemia Using "low-dose" Cyclophosphamide, ATG Plus Fludarabine. Journal of Pediatric Hematology/Oncology. Vol.40, No.4 (2018), e220-e224. doi:10.1097/MPH.0000000000001106 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/47199
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Title
Allogeneic Matched Related Donor Bone Marrow Transplantation for Pediatric Patients with Severe Aplastic Anemia Using "low-dose" Cyclophosphamide, ATG Plus Fludarabine
Abstract
© 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.