聚焦慢性粒细胞白血病  

Spotlight on Chronic Myeloid Leukemia

作　　者：汤森路透 史志祥[2] 张宇辉[2] 

机构地区：[1]不详 [2]中国药科大学

出　　处：《药学进展》2014年第2期150-160,共11页Progress in Pharmaceutical Sciences

摘　　要：随着靶向治疗时代的到来,慢性粒细胞白血病(CML)已经从不治之症转变为基本可控的慢性病。患者生存率有了显著提高,当然在疗效、耐受性及耐药性方面仍有提升的空间。长期以来,酪氨酸激酶抑制剂格列卫(Gleevec)一直被认为是合理药物设计的典范,但更有效的二代药物已经开始作为一线药物获得认可。然而,由于缺乏完整的生存期数据,这些二代药物和格列卫相比所具有的优势还有待于进一步去发现。由于患者需要长时间治疗,毒性和成本的可控性更可能成为选择治疗药物的重要推动因素。治疗慢性粒细胞白血病的产品线首先侧重于解决耐药性问题,尤其是在一线药物治疗失败而三线药物又无法满足需求的情况下。如果患者使用酪氨酸激酶抑制剂有效,那么最终的问题是患者是否可以通过这些药物治愈。With the advent of targeted therapy, chronic myeloid leukemia （CML） has undergone a major transformation from a terminal disease to an essentially manageable chronic condition. Despite significantly improved survival rates, there still remains room for improvement in efficacy, tolerability and resistance potential. Though the tyrosine kinase inhibitor Gleevec is regarded as the poster child for rational drug design, more efficacious second- generation agents are starting to gain acceptance in first-line use; however, in the absence of mature survival data, their hmg-term benefit over Gleevee remains to be seen. As patients remain on therapy tbr increasing periods of time, management of toxicity and cost are likely to be significant drivers of treatment choice. The CML pipeline is primarily tbcused on therapies to overcome resistance, which is particularly problematic following failure of first-line agents and an unreel need still exists fbr third-line options. For patients responding well to treatment with tyrosine kinase inhibitors, the ultimate question is whether such therapy can lead to a cure.

关 键 词：慢性粒细胞白血病 伊马替尼 酪氨酸激酶抑制剂 一线治疗 

分 类 号：R733.7[医药卫生—肿瘤]