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Prepublished online as a Blood First Edition Paper on April 24, 2003; DOI 10.1182/blood-2002-12-3637.

Submitted December 3, 2002
Accepted April 9, 2003
Stem cell transplantation for chronic myeloid leukemia in children
Kate Cwynarski, Irene A G Roberts*, Simona Iacobelli, Anja van Biezen, Ronald Brand, Agnes Devergie, J M Vossen, M Aljurf, W Arcese, F Locatelli, G Dini, D Niethammer, D Niederwieser, and J F Apperley
Department of Haematology, Imperial College Faculty of Medicine, Hammersmith Hospital, London, United Kingdom; Paediatric and Chronic Leukaemia Working Parties of the EBMT
* Corresponding author; email: irene.roberts{at}ic.ac.uk.
Hematopoietic stem cell transplantation (SCT) is the only proven cure for chronic myeloid leukemia (CML), a rare disease in childhood. We report outcome of 314 children with Ph-positive 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) were 66% and 55% (n=314). For 156 children in first chronic phase (CP1) transplanted from HLA-identical siblings OS and LFS were 75% and 63%. For 97 children transplanted in CP1 using VUD SCT 3-year OS and LFS were 65% and 56% reflecting higher transplant-related mortality (TRM) after VUD SCT (35% vs 20%; multivariate HR: 1.9: 95% CI: 1.0-3.5; p=0.05). In a multivariate model for OS and LFS outcome was superior in CP1 versus advanced phase (AP:CP1 OS HR 2.0 [95% CI: 1.3-3]; p=0.001 and LFS HR 1.8 [95% CI: 1.2-2.6]; p=0.003). For relapse, donor source (VUD:sibling HR 0.38 (95% CI: 0.19-0.76; p=0.006) and disease stage were significant (AP: CP1 HR 2.4 (95% CI: 1.36-4.3; p=0.003). This is the first large series to show that SCT confers long-term LFS in the majority of children with CML and helps assess alternative therapy, including tyrosine kinase inhibitors, in children.

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