Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): Results of the EWOG-MDS/EBMT trial

Franco Locatelli, Peter Nöllke, Marco Zecca, Elisabeth Korthof, Edoardo Lanino, Christina Peters, Andrea Pession, Hartmut Kabisch, Cornelio Uderzo, Carmen S. Bonfim, Peter Bader, Dagmar Dilloo, Jan Stary, Alexandra Fischer, Tom Révész, Monika Führer, Henrik Hasle, Monika Trebo, Marry M. Van Den Heuvel-Eibrink, Susanna FenuBrigitte Strahm, Giovanna Giorgiani, Mario Regazzi Bonora, Ulrich Duffner, Charlotte M. Niemeyer

Research output: Contribution to journalArticle


Allogeneic hematopoietic stem cell transplantation (HSCT) is the only proven curative therapy for juvenile myelomonocytic leukemia (JMML). We, the European Working Group on Childhood MDS (EWOG-MDS) and the European Blood and Marrow Transplantation (EBMT) Group, report the outcome of 100 children (67 boys and 33 girls) with JMML given unmanipulated HSCT after a preparative regimen including busulfan, cyclophosphamide, and melphalan. Forty-eight and 52 children received transplants from an HLA-identical relative or an unrelated donor (UD), respectively. The source of hematopoietic stem cells was bone marrow, peripheral blood, and cord blood in 79, 14, and 7 children, respectively. Splenectomy had been performed before HSCT in 24 children. The 5-year cumulative incidence of transplantation-related mortality and leukemia recurrence was 13% and 35%, respectively. Age older than 4 years predicted an increased risk of disease recurrence. The 5-year probability of event-free survival for children given HSCT from either a relative or a UD was 55% and 49%, respectively (P = NS), with median observation time of patients alive being 40 months (range, 6 to 144). In multivariate analysis, age older than 4 years and female sex predicted poorer outcome. Results of this study compare favorably with previously published reports. Disease recurrence remains the major cause of treatment failure. Outcome of UD-HSCT recipients is comparable to that of children receiving transplants from an HLA-identical sibling.

Original languageEnglish
Pages (from-to)410-419
Number of pages10
Issue number1
Publication statusPublished - Jan 1 2005

ASJC Scopus subject areas

  • Hematology

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