Stem cell transplantation for chronic myeloid leukemia in children

Kate Cwynarski, Irene A G Roberts, Simona Iacobelli, Anja Van Biezen, Ronald Brand, Agnes Devergie, Jaak M. Vossen, Mahmoud Aljurf, William Arcese, Franco Locatelli, Giorgio Dini, Dietrich Niethammer, Dietger Niederwieser, Jane F. Apperley

Research output: Contribution to journalArticle


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.

Original languageEnglish
Pages (from-to)1224-1231
Number of pages8
Issue number4
Publication statusPublished - Aug 15 2003

ASJC Scopus subject areas

  • Hematology

Fingerprint Dive into the research topics of 'Stem cell transplantation for chronic myeloid leukemia in children'. Together they form a unique fingerprint.

  • Cite this

    Cwynarski, K., Roberts, I. A. G., Iacobelli, S., Van Biezen, A., Brand, R., Devergie, A., Vossen, J. M., Aljurf, M., Arcese, W., Locatelli, F., Dini, G., Niethammer, D., Niederwieser, D., & Apperley, J. F. (2003). Stem cell transplantation for chronic myeloid leukemia in children. Blood, 102(4), 1224-1231.