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Marrow transplant experience for children with severe aplastic anemia
J E Sanders1, R Storb, C Anasetti
1Department of Pediatrics, Fred Hutchinson Cancer Research Center, Seattle, Washington 98104.
The American Journal of Pediatric Hematology/Oncology
|February 1, 1994
Summary
Combination immunosuppression with methotrexate and cyclosporine significantly improved survival rates for children undergoing allogeneic marrow transplants for severe aplastic anemia by reducing graft versus host disease (GVHD). This approach enhanced event-free survival compared to methotrexate alone.
Area of Science:
- Hematology
- Immunology
- Pediatric Oncology
Background:
- Severe aplastic anemia (SAA) poses significant survival challenges after allogeneic marrow transplant, historically limited by graft rejection and graft-versus-host disease (GVHD).
- Survival rates in the 1970s were approximately 68%, with improvements in the 1980s linked to better GVHD prophylaxis.
Purpose of the Study:
- To evaluate the impact of different GVHD prophylaxis regimens on graft rejection, GVHD incidence, and survival outcomes in pediatric patients with SAA.
- To assess the long-term effects of preparative regimens, including cyclophosphamide (CY), on growth and development in children post-transplant.
Main Methods:
- A retrospective analysis of 140 children (<18 years) with SAA who underwent allogeneic marrow transplantation between 1971 and 1991.
- Comparison of GVHD prophylaxis strategies: methotrexate (MTX) alone versus MTX plus cyclosporine (CSP) for HLA-identical family donor transplants.
- Kaplan-Meier method used to estimate graft rejection, acute and chronic GVHD, survival, and event-free survival (EFS).
Main Results:
- The combination of MTX and CSP significantly improved survival (96%) compared to MTX alone (64%) (p=0.007), though EFS was similar (71% vs. 60%).
- Acute GVHD incidence was lower with MTX plus CSP (11%) versus MTX alone (27%) (p=0.11).
- Children receiving CY only demonstrated normal growth and development, with successful pregnancies reported.
Conclusions:
- High-dose cyclophosphamide (CY) is an effective preparative regimen for pediatric SAA patients with HLA-identical donors.
- Combination GVHD prophylaxis with MTX and CSP significantly enhances survival outcomes.
- Further immunosuppression, like anti-thymocyte globulin, may further reduce graft rejection and improve EFS.