Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial.
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.
Locatelli
Franco
F
Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy. f.locatelli@smatteo.pv.it
Nöllke
Peter
P
Zecca
Marco
M
Korthof
Elisabeth
E
Lanino
Edoardo
E
Peters
Christina
C
Pession
Andrea
A
Kabisch
Hartmut
H
Uderzo
Cornelio
C
Bonfim
Carmen S
CS
Bader
Peter
P
Dilloo
Dagmar
D
Stary
Jan
J
Fischer
Alexandra
A
Révész
Tom
T
Führer
Monika
M
Hasle
Henrik
H
Trebo
Monika
M
van den Heuvel-Eibrink
Marry M
MM
Fenu
Susanna
S
Strahm
Brigitte
B
Giorgiani
Giovanna
G
Bonora
Mario Regazzi
MR
Duffner
Ulrich
U
Niemeyer
Charlotte M
CM
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D007951:Leukemia, Myeloid / Q000276:immunology / Q000473:pathology* / Q000601:surgery*; D008297:Male; D012008:Recurrence; D015996:Survival Rate; D016896:Treatment Outcome