Allogeneic matched related donor bone marrow transplantation for pediatric patients with severe aplastic anemia using "low-dose" cyclophosphamide, ATG plus fludarabine Journal Article
| Authors: | Takpradit, C.; Prockop, S. E.; Kernan, N. A.; Scaradavou, A.; Curran, K.; Ruggiero, J.; Zakak, N.; O'Reilly, R. J.; Boulad, F. |
| Article Title: | Allogeneic matched related donor bone marrow transplantation for pediatric patients with severe aplastic anemia using "low-dose" cyclophosphamide, ATG plus fludarabine |
| Abstract: | 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. © 2018 Wolters Kluwer Health, Inc. All rights reserved. |
| Keywords: | fludarabine; hematopoietic stem cell transplantation; toxicity; aplastic anemia; low-dose cyclophosphamide |
| Journal Title: | Journal of Pediatric Hematology/Oncology |
| Volume: | 40 |
| Issue: | 4 |
| ISSN: | 1077-4114 |
| Publisher: | Lippincott Williams & Wilkins |
| Date Published: | 2018-05-01 |
| Start Page: | e220 |
| End Page: | e224 |
| Language: | English |
| DOI: | 10.1097/mph.0000000000001106 |
| PROVIDER: | scopus |
| PMCID: | PMC5916036 |
| PUBMED: | 29432302 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 1 June 2018 -- Source: Scopus |
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