TY - JOUR
T1 - Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML)
T2 - Results of the EWOG-MDS/EBMT trial
AU - Locatelli, Franco
AU - Nöllke, Peter
AU - Zecca, Marco
AU - Korthof, Elisabeth
AU - Lanino, Edoardo
AU - Peters, Christina
AU - Pession, Andrea
AU - Kabisch, Hartmut
AU - Uderzo, Cornelio
AU - Bonfim, Carmen S.
AU - Bader, Peter
AU - Dilloo, Dagmar
AU - Stary, Jan
AU - Fischer, Alexandra
AU - Révész, Tom
AU - Führer, Monika
AU - Hasle, Henrik
AU - Trebo, Monika
AU - Van Den Heuvel-Eibrink, Marry M.
AU - Fenu, Susanna
AU - Strahm, Brigitte
AU - Giorgiani, Giovanna
AU - Bonora, Mario Regazzi
AU - Duffner, Ulrich
AU - Niemeyer, Charlotte M.
PY - 2005/1/1
Y1 - 2005/1/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=19944428598&partnerID=8YFLogxK
U2 - 10.1182/blood-2004-05-1944
DO - 10.1182/blood-2004-05-1944
M3 - Article
C2 - 15353481
AN - SCOPUS:19944428598
SN - 0006-4971
VL - 105
SP - 410
EP - 419
JO - Blood
JF - Blood
IS - 1
ER -