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Allogeneic Bone Marrow Transplants for Pediatric Severe Aplastic Anemia: Real-world Data comparing Matched Related and Unrelated Donors in a Developing Country. Retrospective study on behalf of the Pediatric Hematopoietic Stem Cell Transplant Working Group of the Brazilian Bone Marrow Transplantation Society (SBTMO) and the Brazil-Seattle Consortium (Gedeco).
- Source :
-
Pediatric transplantation [Pediatr Transplant] 2019 Nov; Vol. 23 (7), pp. e13552. Date of Electronic Publication: 2019 Jul 11. - Publication Year :
- 2019
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Abstract
- In this study, we report on major MRD or URD BMT outcomes in pediatric patients with SAA in Brazil. This was a retrospective study, which included 106 patients ≤18 years old who received a first BMT for SAA. All patients received bone marrow as graft source from an MRD (n = 69) or a URD (n = 37). Conditioning regimen was non-myeloablative in 73.6% of cases, and GVHD prophylaxis comprised a calcineurin inhibitor plus methotrexate in 89.6% of patients. After a median follow-up of 4.5 years after BMT, 81 patients are alive, with a 4-year OS of 77% and no statistically significant difference between the MRD and URD groups (82% vs. 69%, respectively; P = .08). Grade III-IV aGVHD at 6 months and cGVHD at 2 years were observed in 8% and 14% of cases, respectively, and were not statistically different between the groups. Twenty-five (23%) patients died at a median of 2.9 months after BMT. Our study showed that 4-year OS after BMT was not statistically different between MRD and URD recipients. This study shows that the outcomes of pediatric patients transplanted for SAA with a URD in Brazil are approaching those of MRD transplants. In contrast, OS after MRD BMT was lower than we would expect based on previous reports. The wide range of preparatory regimens used by the study centers highlights the need for standardized protocols for these children. Our findings provide a benchmark for future studies focused on improving BMT outcomes in this setting in Brazil.<br /> (© 2019 Wiley Periodicals, Inc.)
- Subjects :
- Adolescent
Brazil
Child
Child, Preschool
Developing Countries
Female
Follow-Up Studies
Humans
Infant
Infant, Newborn
International Cooperation
Male
Retrospective Studies
Severity of Illness Index
Transplantation, Homologous
Treatment Outcome
Washington
Anemia, Aplastic therapy
Bone Marrow Transplantation methods
Unrelated Donors
Subjects
Details
- Language :
- English
- ISSN :
- 1399-3046
- Volume :
- 23
- Issue :
- 7
- Database :
- MEDLINE
- Journal :
- Pediatric transplantation
- Publication Type :
- Academic Journal
- Accession number :
- 31297928
- Full Text :
- https://doi.org/10.1111/petr.13552