Authors: Miharu Yabe Yoshitoshi Ohtsuka Kenichiro Watanabe Jiro Inagaki Nao Yoshida Kazuo Sakashita Harumi Kakuda Hiromasa Yabe Hidemitsu Kurosawa Kazuko Kudo Atsushi Manabe Japanese Pediatric Myelodysplastic Syndrome Study Group
Publish Date: 2014/12/11
Volume: 101, Issue: 2, Pages: 184-190
Abstract
We report the outcomes of 30 patients with juvenile myelomonocytic leukemia JMML who received unmanipulated hematopoietic stem cell transplantation HSCT with oral or intravenous busulfan fludarabine and melphalan between 2001 and 2011 Mutations in PTPN11 were detected in 15 patients Six patients received human leukocyte antigen HLAmatched HSCT from related donors and 24 patients received HSCT from alternative donors including 13 HLAmismatched donors Primary engraftment failed in five patients all of whom had received allografts from HLAmismatched donors HLAmismatched HSCT resulted in poorer eventfree survival than HLAmatched HSCT 288 vs 706 Three patients died of transplantationrelated causes and eight patients experienced hematological relapse including five patients who died due to disease progression Eight patients received a second HSCT and four of these patients have survived The 5year estimated overall survival for all patients was 724 889 for the patients without a mutation in PTPN11 n = 10 and 583 for the patients with a mutation in PTPN11 n = 15 P = 0092 The conditioning regimen reported in the present study achieved hematological and clinical remission in 50 of patients with JMML who received HSCT from alternative donors and may also be effective for JMML patients with PTPN11 mutationThe authors would like to express their gratitude to Dr Yuko Osugi Osaka City General Hospital Dr Chie Kobayashi Ibaraki Children’s Hospital Dr Akira Ohara Toho University School of Medicine Dr Masakatsu Yanagimachi Yokohama City University School of Medicine Dr Naomi Sakata Kinki University School of Medicine Dr Atsushi Ogawa Niigata Cancer Center Dr Asahito Hama Nagoya University School of Medicine Dr Hiroyuki Shimada Keio University School of Medicine Dr Takashi Kaneko Tokyo Metropolitan Children’s Medical Centre Dr Hiroyuki Ishida Matsushita Memorial Hospital and Dr Keiichiro Kawasaki Hyogo Children’s Hospital for their invaluable contribution to the data
Keywords: