TY - JOUR
T1 - Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Children with Juvenile Myelomonocytic Leukemia
T2 - A Report from the Japan Society for Hematopoietic Cell Transplantation
AU - Pediatric Myelodysplastic Syndrome Working Group of the Japan Society for Hematopoietic Cell Transplantation
AU - Yoshida, Nao
AU - Sakaguchi, Hirotoshi
AU - Yabe, Miharu
AU - Hasegawa, Daiichiro
AU - Hama, Asahito
AU - Hasegawa, Daisuke
AU - Kato, Motohiro
AU - Noguchi, Maiko
AU - Terui, Kiminori
AU - Takahashi, Yoshiyuki
AU - Cho, Yuko
AU - Sato, Maho
AU - Koh, Katsuyoshi
AU - Kakuda, Harumi
AU - Shimada, Hiroyuki
AU - Hashii, Yoshiko
AU - Sato, Atsushi
AU - Kato, Koji
AU - Atsuta, Yoshiko
AU - Watanabe, Kenichiro
N1 - Funding Information:
The authors thank all the patients and families, as well as all the physicians and members who provided precise data to the Japan Society for Hematopoietic Cell Transplantation. Financial disclosure: This research was funded by Japanese Red Cross, Nagoya 1st. Hospital Research Grant NFRCH19-0020. This work was supported in part by the Practical Research Project for Allergic Diseases and Immunology (Research Technology of Medical Transplantation) from the Japan Agency for Medical Research and Development , AMED under grant number 19ek0510023h0002 . Conflict of interest statement: There are no conflicts of interest to report. Authorship statement: N.Y. designed the research, organized the project, performed the statistical analysis, analyzed and interpreted the data, and wrote the manuscript; H.Sakaguchi performed the statistical analysis; M.Y., Daiichiro H., A.H., Daisuke H., M.K., and K.W. interpreted the data; M.N., K.T., Y.T., Y.C., M.S., K.Koh, H.K., and H.Shimada gathered and organized the data; Y.H., A.S., K.Kato, and Y.A. had responsibility for development and maintenance of the database. All authors reviewed and approved the final manuscript.
Publisher Copyright:
© 2019 American Society for Transplantation and Cellular Therapy
PY - 2020/5
Y1 - 2020/5
N2 - 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.
AB - 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.
KW - Chronic graft-versus-host disease
KW - Conditioning regimen
KW - Hematopoietic stem cell transplantation
KW - Juvenile myelomonocytic leukemia
UR - http://www.scopus.com/inward/record.url?scp=85077548939&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85077548939&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2019.11.029
DO - 10.1016/j.bbmt.2019.11.029
M3 - Article
C2 - 31790827
AN - SCOPUS:85077548939
SN - 1083-8791
VL - 26
SP - 902
EP - 910
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 5
ER -