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

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.

Original languageEnglish
Pages (from-to)1224-1231
Number of pages8
JournalBlood
Volume102
Issue number4
DOIs
Publication statusPublished - Aug 15 2003

ASJC Scopus subject areas

  • Hematology

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    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. https://doi.org/10.1182/blood-2002-12-3637