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.
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.
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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