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Long‐term results of high‐dose imatinib in children and adolescents with chronic myeloid leukaemia in chronic phase: the Italian experience |
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| Authors: | Fiorina Giona Maria C. Putti Concetta Micalizzi Giuseppe Menna Maria L. Moleti Nicola Santoro Grazia Iaria Saverio Ladogana Roberta Burnelli Caterina Consarino Stefania Varotto Francesca Tucci Chiara Messina Mauro Nanni Daniela Diverio Andrea Biondi Andrea Pession Franco Locatelli Alfonso Piciocchi Enrico Gottardi Giuseppe Saglio Robin Foà |
| Affiliation: | 1. Haematology, Department of Cellular Biotechnologies and Haematology, ‘Sapienza’ University, Rome, Italy;2. Haemato‐Oncology Unit, Department of Women's and Children's Health, University of Padua, Padua, Italy;3. Clinical Experimental Haematology Unit, Gaslini Research Institute of Genoa, Genoa, Italy;4. Paediatric Haematology Unit, Pausilipon Hospital, Naples, Italy;5. Department of Paediatrics, Paediatric Unit ‘F. Vecchio’, University of Bari, Bari, Italy;6. Division of Haematology, ‘Bianchi‐Melacrino‐Morelli’ Hospital, Reggio Calabria, Italy;7. Department of Paediatrics, Haemato‐Oncology Unit, ‘Casa Sollievo della Sofferenza’ Hospital, San Giovanni Rotondo, Italy;8. Paediatric Haemato‐Oncology Unit, Sant'Anna Hospital, Ferrara, Italy;9. Haemato‐Oncology Unit, Azienda Ospedaliera ‘Pugliese‐Ciaccio’, Catanzaro, Italy;10. Department of Paediatrics, University of Milano‐Bicocca, Monza, Italy;11. Haemato‐Oncology Unit, Department of Paediatrics, University of Bologna, Bologna, Italy;12. Department of Paediatrics, University of Pavia, Pavia, Italy;13. Paediatric Haematology‐Oncology, IRCCS Ospedale Paediatrico Bambino Gesù, Rome, Italy;14. Biostatistics Unit, GIMEMA Foundation, Rome, Italy;15. Department of Clinical and Biological Sciences, University of Turin at Orbassano, Turin, Italy |
| 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. |
| Keywords: | chronic myeloid leukaemia childhood imatinib BCR‐ABL1 tyrosine kinase inhibitors |