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Blood, 1 January 2005, Vol. 105, No. 1, pp. 410-419. Prepublished online as a Blood First Edition Paper on September 7, 2004; DOI 10.1182/blood-2004-05-1944. This Article Full Text (PDF) All Versions of this Article: 2004-05-1944v1 105/1/410    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Locatelli, F. Articles by Niemeyer, C. M Search for Related Content PubMed PubMed Citation Articles by Locatelli, F. Articles by Niemeyer, C. M Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted May 25, 2004 Accepted August 3, 2004 Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial Franco Locatelli*, Peter Nollke, 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 Revesz, Monika Fuhrer, Henrik Hasle, Monika Trebo, Marry M van den Heuvel-Eibrink, Susanna Fenu, Brigitte Strahm, Giovanna Giorgiani, Mario R Bonora, Ulrich Duffner, and Charlotte M Niemeyer Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany Department of Pediatric Immunology/Hematology and Stem Cell Transplantation, Leiden University Medical Center, Leiden, The Netherlands Divisione di Ematologia e Oncologia Pediatrica, Istituto G. Gaslini, Genova, Italy St. Anna Kinderspital, Wien, Austria Clinica Pediatrica, Universita di Bologna, Ospedale Sant'Orsola-Malpighi, Bologna, Italy Department of Oncology/Hematology, University Hospital Eppendorf, Hamburg, Germany Clinica Pediatrica, Ospedale Nuovo San Gerardo, Monza, Italy Hospital de Clinicas, Universidade Federal do Parana (UFPR), Curitiba, Brazil University Children's Hospital, University of Tubingen, Tubingen, Germany Department of Pediatric Hematology and Oncology, Heinrich-Heine-University, Dusseldorf, Germany Department of Pediatrics, University Hospital Motol, Prague, Czech Republic Hematology-Oncology Unit, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands von Haunersches, Kinderspital, LMU, Munchen, Germany Department of Pediatrics, Skejby Hospital, Aarhus University, Aarhus, Denmark Dutch Childhood Leukemia Study Group, Erasmus University Medical Center- Sophia Children's Hospital, Rotterdam, The Netherlands Dipartimento di Ematologia, Universita La Sapienza, Roma, Italy Dipartimento di Farmacologia Clinica, IRCCS Policlinico San Matteo, Pavia, Italy * Corresponding author; email: f.locatelli{at}smatteo.pv.it. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only proven curative therapy for juvenile myelomonocytic leukemia (JMML). We report the outcome of 100 children (67 males and 33 females) with JMML given unmanipulated HSCT after a preparative regimen including busulfan, cyclophosphamide and melphalan. Forty-eight and 52 children were transplanted from an HLA-identical relative or an unrelated donor (UD), respectively. 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 transplant-related mortality and leukemia recurrence were 13% and 35%, respectively. Age greater 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 an UD was 55% and 49%, respectively (p=NS), median observation time of patients alive being 40 months (range 6-144 months). In multivariate analysis, age greater 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 transplanted from an HLA-identical sibling. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: Z. Yang, T. Kondo, C. S. Voorhorst, S. C. Nabinger, L. Ndong, F. Yin, E. M. Chan, M. Yu, O. Wurstlin, C. P. Kratz, et al. Increased c-Jun Expression and Reduced GATA2 Expression Promote Aberrant Monocytic Differentiation Induced by Activating PTPN11 Mutants Mol. Cell. 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