PMID- 25504334
OWN - NLM
STAT- MEDLINE
DCOM- 20150818
LR  - 20181113
IS  - 1865-3774 (Electronic)
IS  - 0925-5710 (Linking)
VI  - 101
IP  - 2
DP  - 2015 Feb
TI  - Transplantation for juvenile myelomonocytic leukemia: a retrospective study of 30 
      children treated with a regimen of busulfan, fludarabine, and melphalan.
PG  - 184-90
LID - 10.1007/s12185-014-1715-7 [doi]
AB  - 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 (HLA)-matched HSCT from related donors, and 24 patients 
      received HSCT from alternative donors, including 13 HLA-mismatched donors. 
      Primary engraftment failed in five patients, all of whom had received allografts 
      from HLA-mismatched donors. HLA-mismatched HSCT resulted in poorer event-free 
      survival than HLA-matched HSCT (28.8 vs. 70.6 %). Three patients died of 
      transplantation-related 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 
      5-year estimated overall survival for all patients was 72.4: 88.9 % for the 
      patients without a mutation in PTPN11 (n = 10) and 58.3 % for the patients with a 
      mutation in PTPN11 (n = 15) (P = 0.092). 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 mutation.
FAU - Yabe, Miharu
AU  - Yabe M
AD  - Department of Cell Transplantation and Regenerative Medicine, Tokai University 
      Hospital, 143 Shimokasuya, Isehara, Kanagawa, 259-1193, Japan, 
      miharu@is.icc.u-tokai.ac.jp.
FAU - Ohtsuka, Yoshitoshi
AU  - Ohtsuka Y
FAU - Watanabe, Kenichiro
AU  - Watanabe K
FAU - Inagaki, Jiro
AU  - Inagaki J
FAU - Yoshida, Nao
AU  - Yoshida N
FAU - Sakashita, Kazuo
AU  - Sakashita K
FAU - Kakuda, Harumi
AU  - Kakuda H
FAU - Yabe, Hiromasa
AU  - Yabe H
FAU - Kurosawa, Hidemitsu
AU  - Kurosawa H
FAU - Kudo, Kazuko
AU  - Kudo K
FAU - Manabe, Atsushi
AU  - Manabe A
CN  - Japanese Pediatric Myelodysplastic Syndrome Study Group
LA  - eng
PT  - Journal Article
DEP - 20141211
PL  - Japan
TA  - Int J Hematol
JT  - International journal of hematology
JID - 9111627
RN  - FA2DM6879K (Vidarabine)
RN  - G1LN9045DK (Busulfan)
RN  - P2K93U8740 (fludarabine)
RN  - Q41OR9510P (Melphalan)
SB  - IM
MH  - Antineoplastic Combined Chemotherapy Protocols/adverse effects/therapeutic use
MH  - Busulfan/administration & dosage
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - Graft vs Host Disease/etiology/prevention & control
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects
MH  - Humans
MH  - Infant
MH  - Infant, Newborn
MH  - Leukemia, Myelomonocytic, Juvenile/complications/mortality/*therapy
MH  - Male
MH  - Melphalan/administration & dosage
MH  - Retreatment
MH  - Retrospective Studies
MH  - Transplantation Conditioning
MH  - Transplantation, Homologous
MH  - Treatment Outcome
MH  - Vidarabine/administration & dosage/analogs & derivatives
EDAT- 2014/12/17 06:00
MHDA- 2015/08/19 06:00
CRDT- 2014/12/16 06:00
PHST- 2014/08/02 00:00 [received]
PHST- 2014/11/27 00:00 [accepted]
PHST- 2014/11/21 00:00 [revised]
PHST- 2014/12/16 06:00 [entrez]
PHST- 2014/12/17 06:00 [pubmed]
PHST- 2015/08/19 06:00 [medline]
AID - 10.1007/s12185-014-1715-7 [doi]
PST - ppublish
SO  - Int J Hematol. 2015 Feb;101(2):184-90. doi: 10.1007/s12185-014-1715-7. Epub 2014 
      Dec 11.