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台湾多中心经验:一线伊马替尼治疗的慢性髓性白血病儿童的分子反应较慢,但与成人相比生存相当。

Children with chronic myeloid leukaemia treated with front-line imatinib have a slower molecular response and comparable survival compared with adults: a multicenter experience in Taiwan.

机构信息

Department of Hematology-Oncology, MacKay Children's Hospital and MacKay Medical College, Taipei, Taiwan.

Division of Hematology-Oncology, Chang Gung Memorial Hospital-Linkou, Taoyuan, Taiwan.

出版信息

Br J Cancer. 2023 Mar;128(7):1294-1300. doi: 10.1038/s41416-023-02162-9. Epub 2023 Jan 30.

Abstract

BACKGROUND

The direct comparison of molecular responses of front-line imatinib (IM) monitored at the same laboratory between children and adults with chronic phase (CP) of chronic myeloid leukaemia (CML) had not been reported. In this multicenter study, we compared the landmark molecular responses and outcomes of paediatric and adult CML-CP cohorts treated with front-line IM in whom the BCR::ABL1 transcript levels were monitored at the same accredited laboratory in Taiwan.

METHODS

Between June 2004 and July 2020, 55 newly diagnosed paediatric and 782 adult CML-CP patients, with molecular diagnosis and monitoring at the same reference laboratory in Taiwan, were enrolled. The criteria of 2020 European LeukemiaNet were applied to evaluate the molecular responses.

RESULTS

By year 5, the cumulative incidences of IS <1%, MMR, MR and MR of paediatric patients were all significantly lower than those of adult patients (58 vs 75%, 48 vs 66%, 25 vs 44%, 16 vs 34%, respectively). The 10-year progression-free survival (PFS) (90%) and overall survival (OS) (94%) of paediatric patients did not differ from those (92%) of adult patients.

CONCLUSIONS

We demonstrated the paediatric cohort had slower molecular responses to front-line IM and similar outcomes in 10-year PFS and OS in real-world practice.

摘要

背景

尚未有研究直接比较过在同一实验室监测的儿童和成人慢性髓系白血病(CML)慢性期一线伊马替尼(IM)的分子反应。在这项多中心研究中,我们比较了台湾同一认证实验室监测的一线 IM 治疗的儿科和成人 CML-CP 队列的标志性分子反应和结果。

方法

2004 年 6 月至 2020 年 7 月,55 例新诊断的儿科和 782 例成人 CML-CP 患者在台湾的同一参考实验室进行了分子诊断和监测。采用 2020 年欧洲白血病网的标准评估分子反应。

结果

5 年内,儿科患者的 IS<1%、MMR、MR 和 MR 累积发生率均明显低于成年患者(分别为 58% vs 75%、48% vs 66%、25% vs 44%、16% vs 34%)。儿科患者的 10 年无进展生存(PFS)(90%)和总生存(OS)(94%)与成年患者(92%)无差异。

结论

我们在真实世界实践中证明了儿科患者一线 IM 治疗的分子反应较慢,但在 10 年 PFS 和 OS 方面的结果相似。

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