Giona Fiorina, Putti Maria C, Micalizzi Concetta, Menna Giuseppe, Moleti Maria L, Santoro Nicola, Iaria Grazia, Ladogana Saverio, Burnelli Roberta, Consarino Caterina, Varotto Stefania, Tucci Francesca, Messina Chiara, Nanni Mauro, Diverio Daniela, Biondi Andrea, Pession Andrea, Locatelli Franco, Piciocchi Alfonso, Gottardi Enrico, Saglio Giuseppe, Foà Robin
Haematology, Department of Cellular Biotechnologies and Haematology, 'Sapienza' University, Rome, Italy.
Haemato-Oncology Unit, Department of Women's and Children's Health, University of Padua, Padua, Italy.
Br J Haematol. 2015 Aug;170(3):398-407. doi: 10.1111/bjh.13453. Epub 2015 Apr 20.
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.
甲磺酸伊马替尼（IM）用于治疗儿童慢性髓性白血病（CML）。IM的最有效剂量及其在儿童中的长期疗效尚未明确界定。这项多中心研究的目的是报告诊断时年龄＜18岁的慢性期CML（CP-CML）患者接受高剂量IM（340mg/m²/d）治疗的长期结果。共有47例诊断时中位年龄为11岁9个月的CP-CML患者在意大利的9个中心入组。91.5%的可评估患者在中位时间6个月时实现了完全细胞遗传学缓解。12个月时BCR-ABL1国际量表≤0.1%（主要分子反应；MMR）和≤0.01%（分子反应；MR）的患者分别为66.6%和33%。在随访期间，分别有78.6%和61%的儿童实现了MMR和MR。3例长期接受治疗且分子反应持久的儿童安全停用了IM。12例患者（8例细胞遗传学/分子反应者）接受了干细胞移植。继续接受IM治疗的反应性患者和接受移植的患者在96个月时的无进展生存概率分别为60%和50%。中位随访52个月（范围3-146个月）后，所有患者均存活。高剂量IM对患有CP-CML的儿童和青少年是一种长期有效的治疗方法。
