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儿童慢性髓性白血病(CML)的预后预测。

Outcome prediction of chronic myeloid leukemia (CML) in children.

机构信息

Department of Paediatrics and Adolescent Medicine, Hong Kong Children's Hospital, Ngau Tau Kok, Hong Kong.

Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, Pok Fu Lam, Hong Kong.

出版信息

Ann Hematol. 2022 Aug;101(8):1677-1688. doi: 10.1007/s00277-022-04852-5. Epub 2022 Jun 1.

Abstract

We evaluated the feasibility of existing risk assessment tools for chronic myeloid leukemia (CML) in children. Fifty-five patients with newly diagnosed CML between 1996 and 2019 were included. Forty-nine patients presented in chronic phase, thirty-six of whom were treated with upfront tyrosine kinase inhibitor (CP-TKI group); one presented in accelerated phase and four in blastic phase. Treatment, survival, responses, and tolerance were evaluated. All patients in the CP-TKI group received imatinib as their first TKI treatment. The 10-year overall survival (OS), progression-free survival (PFS), and event-free survival (EFS) of TKI-treated group was 97%, 91.4%, and 72.3%, respectively. At 60 months, the rates of major molecular response were 81.2% and deep molecular response was 67.5%. The EUTOS long-term survival (ELTS) risk grouping did not predict OS, PFS, or EFS. The IMAFAIL risk groups were correlated with the risk of imatinib failure. Further studies are required to modify the existing risk assessment tools for children.

摘要

我们评估了现有的慢性髓性白血病(CML)儿童风险评估工具的可行性。纳入了 1996 年至 2019 年间新诊断为 CML 的 55 例患者。49 例患者处于慢性期,其中 36 例接受酪氨酸激酶抑制剂(CP-TKI 组)治疗;1 例加速期,4 例急变期。评估了治疗、生存、反应和耐受性。CP-TKI 组所有患者均接受伊马替尼作为其首个 TKI 治疗。TKI 治疗组的 10 年总生存率(OS)、无进展生存率(PFS)和无事件生存率(EFS)分别为 97%、91.4%和 72.3%。60 个月时,主要分子反应率为 81.2%,深度分子反应率为 67.5%。EUTOS 长期生存(ELTS)风险分组不能预测 OS、PFS 或 EFS。IMAAFALL 风险组与伊马替尼失败的风险相关。需要进一步研究来修改现有的儿童风险评估工具。

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