聚焦慢性粒细胞白血病

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。

Spotlight on Chronic Myeloid Leukemia

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 secondgeneration agents are starting to gain acceptance in first-line use; however, in the absence of mature survival data, their long-term benefit over Gleevec remains to be seen. As patients remain on therapy for increasing periods of time, management of toxicity and cost are likely to be significant drivers of treatment choice. The CML pipeline is primarily focused on therapies to overcome resistance, which is particularly problematic following failure of first-line agents and an unmet need still exists for 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.