title: A conditioning regimen of busulfan, fludarabine, and melphalan for allogeneic stem cell transplantation in children with juvenile myelomonocytic leukemia.
abstract: A pilot study was undertaken using a myeloablative conditioning with fludarabine, busulfan, and melphalan to improve the outcome of HSCT in 10 children, aged six months to six yr, with JMML. All patients were conditioned with oral busulfan (560 mg/m(2)), fludarabine (120 mg/m(2)), and melphalan (180-210 mg/m(2)) prior to HSCT, and received stem cells from bone marrow in seven cases, and from cord blood in three cases. Engraftment was documented in eight patients, whereas graft failure occurred in two, one of whom had received HLA-mismatched cord blood and other had received bone marrow from HLA-mismatched mother. Three patients, including two in who graft failure had occurred, relapsed. Five patients developed acute GVHD and two developed chronic GVHD. Seven patients are alive and in remission 27-69 months after transplantation. Thus, our study showed that HSCT following conditioning with fludarabine, busulfan, and melphalan was well tolerated and appeared to be effective for JMML.
authors.affiliation: Department of Cell Transplantation, Tokai University School of Medicine, Kanagawa, Japan. miharu@is.icc.u-tokai.ac.jp
authors.forename: Miharu
authors.initials: M
authors.lastname: Yabe
authors.forename: Masahiro
authors.initials: M
authors.lastname: Sako
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Yuko
authors.initials: Y
authors.lastname: Osugi
authors.forename: Hidemitsu
authors.initials: H
authors.lastname: Kurosawa
authors.forename: Taemi
authors.initials: T
authors.lastname: Nara
authors.forename: Mika
authors.initials: M
authors.lastname: Tokuyama
authors.forename: Souichi
authors.initials: S
authors.lastname: Adachi
authors.forename: Chie
authors.initials: C
authors.lastname: Kobayashi
authors.forename: Masakatsu
authors.initials: M
authors.lastname: Yanagimachi
authors.forename: Yoshitoshi
authors.initials: Y
authors.lastname: Ohtsuka
authors.forename: Yozo
authors.initials: Y
authors.lastname: Nakazawa
authors.forename: Chitose
authors.initials: C
authors.lastname: Ogawa
authors.forename: Atsushi
authors.initials: A
authors.lastname: Manabe
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
authors.forename: Tatsutoshi
authors.initials: T
authors.lastname: Nakahata
mesh_terms: D000284:Administration, Oral; D002066:Busulfan / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D005260:Female; D049690:History, Ancient; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D054429:Leukemia, Myelomonocytic, Juvenile / Q000188:drug therapy*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D010865:Pilot Projects; D012074:Remission Induction; D033581:Stem Cell Transplantation / Q000379:methods*; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous / Q000379:methods; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives*
chemical_list: D007166:Immunosuppressive Agents; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008558:Melphalan