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伊马替尼治疗儿童慢性粒细胞白血病的疗效与安全性——一项单中心研究

Efficacy and Safety of Imatinib in Paediatric CML - A Single Centre Study.

作者信息

Smeding Cynthia, Szydło Aleksandra, Pieluszczak Krzysztof, Grzeszkiewicz Katarzyna, Pawelec Katarzyna

机构信息

Student Scientific Group 'SPHEROCYTE', Department of Paediatric Haematology and Oncology, Medical University of Warsaw, Warsaw, Poland.

Department of Paediatric Haematology and Oncology, Medical University of Warsaw, Warsaw, Poland.

出版信息

In Vivo. 2019 May-Jun;33(3):869-875. doi: 10.21873/invivo.11552.

DOI:10.21873/invivo.11552
PMID:31028210
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6559902/
Abstract

BACKGROUND/AIM: Chronic myeloid leukaemia (CML) rarely affects the paediatric population and has an incidence of 0.06-0.12/100,000 children per year. The dire clinical course of paediatric CML is further exacerbated by the adverse effects of long-term imatinib therapy.

PATIENTS AND METHODS

Our cohort comprised 14 CML patients who were treated with imatinib between July 2010 and September 2018. The European Leukaemia Net (ELN) standard milestones of response criteria were used to evaluate its therapeutic effectiveness.

RESULTS

Complete haematological remission and partial cytogenetic response were achieved in all patients. Complete cytogenetic response was achieved in seven patients. Major molecular response was achieved in six patients. Two patients underwent haematopoietic stem cell transplantation due to unsatisfactory response to imatinib.

CONCLUSION

Imatinib is effective in treating paediatric CML and limits the progression to advanced stages, however, the quality of life still needs to be optimised.

摘要

背景/目的:慢性髓性白血病(CML)很少影响儿童群体,每年发病率为每10万名儿童中有0.06 - 0.12例。长期伊马替尼治疗的不良反应进一步加剧了儿童CML的严重临床病程。

患者与方法

我们的队列包括2010年7月至2018年9月期间接受伊马替尼治疗的14例CML患者。采用欧洲白血病网(ELN)标准反应标准里程碑来评估其治疗效果。

结果

所有患者均实现完全血液学缓解和部分细胞遗传学反应。7例患者实现完全细胞遗传学反应。6例患者实现主要分子反应。2例患者因对伊马替尼反应不佳而接受造血干细胞移植。

结论

伊马替尼在治疗儿童CML方面有效,并限制其进展至晚期阶段,然而,生活质量仍需优化。

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Leukemia. 2018 Jul;32(7):1657-1669. doi: 10.1038/s41375-018-0179-9. Epub 2018 Jun 20.
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Imatinib in the treatment of chronic myeloid leukemia in children and adolescents is effective and well tolerated: Report of the Polish Pediatric Study Group for the Treatment of Leukemias and Lymphomas.伊马替尼治疗儿童和青少年慢性粒细胞白血病疗效显著且耐受性良好:波兰儿童白血病和淋巴瘤治疗研究组报告
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Pharmacology and pharmacokinetics of imatinib in pediatric patients.儿童患者伊马替尼的药理学和药代动力学。
Expert Rev Clin Pharmacol. 2018 Mar;11(3):219-231. doi: 10.1080/17512433.2018.1398644. Epub 2017 Nov 6.
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Deeper molecular response is a predictive factor for treatment-free remission after imatinib discontinuation in patients with chronic phase chronic myeloid leukemia: the JALSG-STIM213 study.深度分子反应是慢性期慢性髓性白血病患者停用伊马替尼后无治疗缓解的预测因素:日本成人白血病研究组-STIM213研究
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