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 (340 mg/m2/d) in CML patients in chronic phase (CP-CML) aged <18 years at diagnosis. A total of 47 CP-CML patients with a median age at diagnosis of 11 years 9 months were enrolled in nine Italian centres. Complete cytogenetic response was achieved in 91·5% of the evaluable patients at a median time of 6 months. BCR-ABL1 International Scale ≤ 0·1% (major molecular response; MMR) and ≤0·01% (molecular response; MR) at 12 months were 66·6% and 33%, respectively. During follow-up, MMR and MR were achieved in 78·6% 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 96 months for responding patients who continued IM and for those transplanted were 60% and 50%, respectively. After a median follow-up of 52 months (range 3–146), all patients are alive. High-dose IM is a long-term effective therapy in children and adolescents with CP-CML.

Giona, F., Putti, M., Micalizzi, C., Menna, G., Moleti, M., Santoro, N., et al. (2015). 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, 170(3), 398-407 [10.1111/bjh.13453].

Long-term results of high-dose imatinib in children and adolescents with chronic myeloid leukaemia in chronic phase: The Italian experience

BIONDI, ANDREA;
2015

Abstract

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 (340 mg/m2/d) in CML patients in chronic phase (CP-CML) aged <18 years at diagnosis. A total of 47 CP-CML patients with a median age at diagnosis of 11 years 9 months were enrolled in nine Italian centres. Complete cytogenetic response was achieved in 91·5% of the evaluable patients at a median time of 6 months. BCR-ABL1 International Scale ≤ 0·1% (major molecular response; MMR) and ≤0·01% (molecular response; MR) at 12 months were 66·6% and 33%, respectively. During follow-up, MMR and MR were achieved in 78·6% 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 96 months for responding patients who continued IM and for those transplanted were 60% and 50%, respectively. After a median follow-up of 52 months (range 3–146), all patients are alive. High-dose IM is a long-term effective therapy in children and adolescents with CP-CML.
Articolo in rivista - Articolo scientifico
BCR-ABL1; Childhood; Chronic myeloid leukaemia; Imatinib; Tyrosine kinase inhibitors; Adolescent; Antineoplastic Agents; Benzamides; Child; Child, Preschool; Disease-Free Survival; Female; Follow-Up Studies; Fusion Proteins, bcr-abl; Humans; Imatinib Mesylate; Italy; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Male; Piperazines; Pyrimidines; Stem Cell Transplantation; Survival Rate; Hematology
English
398
407
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Giona, F., Putti, M., Micalizzi, C., Menna, G., Moleti, M., Santoro, N., et al. (2015). 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, 170(3), 398-407 [10.1111/bjh.13453].
Giona, F; Putti, M; Micalizzi, C; Menna, G; Moleti, M; Santoro, N; Iaria, G; Ladogana, S; Burnelli, R; Consarino, C; Varotto, S; Tucci, F; Messina, C; Nanni, M; Diverio, D; Biondi, A; Pession, A; Locatelli, F; Piciocchi, A; Gottardi, E; Saglio, G; Foà, R
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/97384
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