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Allogeneic Matched Related Donor Bone Marrow Transplantation for Pediatric Patients With Severe Aplastic Anemia Using 'Low-dose' Cyclophosphamide, ATG Plus Fludarabine
- Source :
- Journal of Pediatric Hematology/Oncology. 40:e220-e224
- Publication Year :
- 2018
- Publisher :
- Ovid Technologies (Wolters Kluwer Health), 2018.
-
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×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.
- Subjects :
- Adult
Male
medicine.medical_specialty
Adolescent
Cyclophosphamide
Anemia
Severity of Illness Index
Gastroenterology
Disease-Free Survival
Article
03 medical and health sciences
0302 clinical medicine
Internal medicine
medicine
Humans
Aplastic anemia
Child
Survival rate
Antilymphocyte Serum
Bone Marrow Transplantation
business.industry
Graft Survival
Anemia, Aplastic
Hematology
Allografts
medicine.disease
Tissue Donors
Fludarabine
Survival Rate
Transplantation
Regimen
surgical procedures, operative
Graft-versus-host disease
Oncology
030220 oncology & carcinogenesis
Pediatrics, Perinatology and Child Health
Female
business
Vidarabine
Follow-Up Studies
030215 immunology
medicine.drug
Subjects
Details
- ISSN :
- 10774114
- Volume :
- 40
- Database :
- OpenAIRE
- Journal :
- Journal of Pediatric Hematology/Oncology
- Accession number :
- edsair.doi.dedup.....d3b666961e9249d76b8083c7b6193412