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 LIFS, outcomes were superior in CP1 than in advanced phase (AP/CP1) (OS HR, 2.0; 95% CI, 1.3-3; P = .001; LFS HIR, 1.8; 96% CI, 1.2-2.6; P = .003). For relapse, donor source (VUD/ sibling) (HR, 0.381- 95% CI, 0.19-0.76; P = .006) and disease stage (AP/CP1) (HR, 2.4; 95% Cl, 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. (C) 2003 by The American Society of Hematology.

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., Stem cell transplantation for chronic myeloid leukemia in children, <<BLOOD>>, 2003; 102 (4): 1224-1231. [doi:10.1182/blood-2002-12-3637] [https://hdl.handle.net/10807/262010]

Stem cell transplantation for chronic myeloid leukemia in children

Locatelli, Franco;
2003

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 LIFS, outcomes were superior in CP1 than in advanced phase (AP/CP1) (OS HR, 2.0; 95% CI, 1.3-3; P = .001; LFS HIR, 1.8; 96% CI, 1.2-2.6; P = .003). For relapse, donor source (VUD/ sibling) (HR, 0.381- 95% CI, 0.19-0.76; P = .006) and disease stage (AP/CP1) (HR, 2.4; 95% Cl, 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. (C) 2003 by The American Society of Hematology.
2003
Inglese
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., Stem cell transplantation for chronic myeloid leukemia in children, <<BLOOD>>, 2003; 102 (4): 1224-1231. [doi:10.1182/blood-2002-12-3637] [https://hdl.handle.net/10807/262010]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/262010
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