title: Stem cell transplantation for chronic myeloid leukemia in children.
abstract: Hematopoietic stem cell transplantation (SCT) is the only proven cure for chronic myeloid leukemia (CML), a rare disease in childhood. We report outcomes of 314 children with Philadelphia-chromosome-positive (Ph+) CML undergoing SCT from HLA-matched siblings (n = 182) or volunteer-unrelated donors (VUD; n = 132). Three-year overall survival (OS) and leukemia-free survival (LFS) rates were 66% and 55% (n = 314). For 156 children in first chronic phase (CP1) who underwent transplantation from HLA-identical siblings, OS and LFS rates were 75% and 63%. For 97 children who underwent SCT in CP1 from VUD, 3-year OS and LFS rates were 65% and 56%, reflecting higher transplantation-related mortality (TRM) after VUD SCT (35% vs 20%; multivariate hazard ratio [HR], 1.9; 95% confidence interval [CI], 1.0-3.5; P =.05). In a multivariate model for OS and LFS, outcomes were superior in CP1 than in advanced phase (AP/CP1) (OS HR, 2.0; 95% CI, 1.3-3; P =.001; LFS HR, 1.8; 95% CI, 1.2-2.6; P =.003). For relapse, donor source (VUD/sibling) (HR, 0.38; 95% CI, 0.19-0.76; P =.006) and disease stage (AP/CP1) (HR, 2.4; 95% CI, 1.36-4.3; P =.003) were significant. This is the first large series to show that SCT confers long-term LFS in most children with CML and helps assess alternative therapy, including tyrosine kinase inhibitors.
authors.affiliation: Paediatric and Chronic Leukaemia Working Parties of the European Group for Blood and Marrow Transplantation.
authors.forename: Kate
authors.initials: K
authors.lastname: Cwynarski
authors.forename: Irene A G
authors.initials: IA
authors.lastname: Roberts
authors.forename: Simona
authors.initials: S
authors.lastname: Iacobelli
authors.forename: Anja
authors.initials: A
authors.lastname: van Biezen
authors.forename: Ronald
authors.initials: R
authors.lastname: Brand
authors.forename: Agnes
authors.initials: A
authors.lastname: Devergie
authors.forename: Jaak M
authors.initials: JM
authors.lastname: Vossen
authors.forename: Mahmoud
authors.initials: M
authors.lastname: Aljurf
authors.forename: William
authors.initials: W
authors.lastname: Arcese
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Giorgio
authors.initials: G
authors.lastname: Dini
authors.forename: Dietrich
authors.initials: D
authors.lastname: Niethammer
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: Jane F
authors.initials: JF
authors.lastname: Apperley
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D003587:Cytomegalovirus; D003586:Cytomegalovirus Infections / Q000097:blood; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D016898:Interferon-alpha / Q000627:therapeutic use; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000628:therapy* / Q000821:virology; D008297:Male; D015999:Multivariate Analysis; D016016:Proportional Hazards Models; D035781:Siblings; D015996:Survival Rate; D014019:Tissue Donors; D016896:Treatment Outcome
chemical_list: D016898:Interferon-alpha