Long-term results of high-dose imatinib in children and adolescents with chronic myeloid leukaemia in chronic phase: the Italian experience.
BRITISH JOURNAL OF HAEMATOLOGY(2015)
摘要
Imatinib mesylate (IM) is used for the management of childhood chronic myeloid leukaemia (CML). The most effective dosage of IM and its long-term efficacy in children are not well defined. The purpose of this multicentre study is to report on the long-term results of high-dose IM (340mg/m(2)/d) in CML patients in chronic phase (CP-CML) aged <18years at diagnosis. A total of 47 CP-CML patients with a median age at diagnosis of 11years 9months were enrolled in nine Italian centres. Complete cytogenetic response was achieved in 915% of the evaluable patients at a median time of 6months. BCR-ABL1 International Scale01% (major molecular response; MMR) and 001% (molecular response; MR) at 12months were 666% and 33%, respectively. During follow-up, MMR and MR were achieved in 786% and 61% of children, respectively. IM was safely discontinued in 3 long-term treated children with a durable MR. Twelve patients (eight cytogenetic/molecular responders) underwent stem cell transplantation. The progression-free survival probabilities at 96months for responding patients who continued IM and for those transplanted were 60% and 50%, respectively. After a median follow-up of 52months (range 3-146), all patients are alive. High-dose IM is a long-term effective therapy in children and adolescents with CP-CML.
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关键词
chronic myeloid leukaemia,childhood,imatinib,BCR-ABL1,tyrosine kinase inhibitors
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