Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Children with Juvenile Myelomonocytic Leukemia: A Report from the Japan Society for Hematopoietic Cell Transplantation |
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| Affiliation: | 1. Department of Hematology and Oncology, Children''s Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan;2. Department of Innovative Medical Science, Tokai University School of Medicine, Isehara, Japan;3. Departments of Hematology and Oncology, Kobe Children''s Hospital, Kobe, Japan;4. Department of Pediatrics, St. Luke''s International Hospital, Tokyo, Japan;5. Children''s Cancer Center, National Center for Child Health and Development, Tokyo, Japan;6. Department of Pediatrics, National Kyushu Cancer Center, Fukuoka, Japan;7. Department of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan;8. Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan;9. Department of Pediatrics, Hokkaido University Hospital, Sapporo, Japan;10. Department of Hematology/Oncology, Osaka Women''s and Children''s Hospital, Izumi, Japan;11. Department of Hematology/Oncology, Saitama Children''s Medical Center, Saitama, Japan;12. Department of Hematology/Oncology, Chiba Children''s Hospital, Chiba, Japan;13. Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan;14. Department of Cancer Immunotherapy, Osaka University Graduate School of Medicine, Osaka, Japan;15. Departments of Hematology and Oncology, Miyagi Children''s Hospital, Sendai, Japan;16. Central Japan Cord Blood Bank, Seto, Japan;17. Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan;18. Department of Healthcare Administration, Nagoya University Graduate School of Medicine, Nagoya, Japan;19. Department of Hematology and Oncology, Shizuoka Children''s Hospital, Shizuoka, Japan |
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| Abstract: | Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for juvenile myelomonocytic leukemia (JMML), but few large studies of HSCT for JMML exist. Using data from the Japan Society for Hematopoietic Cell Transplantation registry, we analyzed the outcomes of 129 children with JMML who underwent HSCT between 2000 and 2011. The 5-year overall survival (OS) rate and cumulative incidence of relapse were 64% and 34%, respectively. A regimen of busulfan/fludarabine/melphalan was the most commonly used (59 patients) and provided the best outcomes; the 5-year OS rate reached 73%, and the cumulative incidences of relapse and transplantation-related mortality were 26% and 9%, respectively. In contrast, the use of the irradiation-based myeloablative regimen was the most significant risk factor for OS (hazard ratio HR], 2.92; P = .004) in the multivariate model. In addition, chronic graft-versus-host disease (GVHD) was strongly associated with lower relapse (HR, 0.37; P = .029) and favorable survival (HR, 0.22; P = .006). The current study has shown that a significant proportion of children with JMML can be cured with HSCT, especially those receiving the busulfan/fludarabine/melphalan regimen. Based on the lower relapse and better survival observed in patients with chronic GVHD, additional treatment strategies that focus on enhancing graft-versus-leukemia effects may further improve survival. |
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