Transplantation for juvenile myelomonocytic leukemia: a retrospective study of 30 children treated with a regimen of busulfan, fludarabine, and melphalan

Miharu Yabe, Yoshitoshi Ohtsuka, Kenichiro Watanabe, Jiro Inagaki, Nao Yoshida, Kazuo Sakashita, Harumi Kakuda, Hiromasa Yabe, Hidemitsu Kurosawa, Kazuko Kudo, Atsushi Manabe

Research output: Contribution to journalArticle

17 Citations (Scopus)

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 (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.

Original languageEnglish
Pages (from-to)184-190
Number of pages7
JournalInternational Journal of Hematology
Volume101
Issue number2
DOIs
Publication statusPublished - 07-02-2015
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Hematology

Fingerprint Dive into the research topics of 'Transplantation for juvenile myelomonocytic leukemia: a retrospective study of 30 children treated with a regimen of busulfan, fludarabine, and melphalan'. Together they form a unique fingerprint.

  • Cite this

    Yabe, M., Ohtsuka, Y., Watanabe, K., Inagaki, J., Yoshida, N., Sakashita, K., Kakuda, H., Yabe, H., Kurosawa, H., Kudo, K., & Manabe, A. (2015). Transplantation for juvenile myelomonocytic leukemia: a retrospective study of 30 children treated with a regimen of busulfan, fludarabine, and melphalan. International Journal of Hematology, 101(2), 184-190. https://doi.org/10.1007/s12185-014-1715-7