Cancer Theme, South Australian Health and Medical Research Institute, Adelaide, South Australia, Australia.
School of Medicine, University of Adelaide, Adelaide, South Australia, Australia.
Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):168-176. doi: 10.1182/asheducation-2018.1.168.
With the advent of tyrosine kinase inhibitors (TKIs), the goals of therapy in chronic myeloid leukemia (CML) are steadily shifting. Long-term disease control on TKI therapy has been the goal and expectation for most patients. More recently, treatment-free remission (TFR) has entered mainstream practice and is increasingly being adopted as the main goal of therapy. This therapeutic shift not only influences TKI selection but also, has necessitated the refinement and dissemination of highly sensitive and accurate molecular monitoring techniques. Measurement of BCR-ABL1 messenger RNA expression through reverse transcription quantitative polymerase chain reaction, reported according to the International Scale, has become the primary tool for response assessment in CML. Achieving specific time-dependent molecular milestones, as defined by global therapeutic guidelines, has been established as critical in maximizing optimal outcomes while identifying patients at risk of therapy failure. Depth and duration of a deep molecular response have become the new therapeutic targets in patients considered for TFR. Consequently, molecular monitoring in CML has become even more critical to ongoing response assessment, identifying patients with TKI resistance and poor drug adherence, and enabling TFR to be attempted safely and effectively.
随着酪氨酸激酶抑制剂（TKI）的出现，慢性髓性白血病（CML）的治疗目标正在稳步转变。TKI 治疗的长期疾病控制一直是大多数患者的目标和期望。最近，无治疗缓解（TFR）已进入主流实践，并越来越多地被作为主要治疗目标。这种治疗转变不仅影响 TKI 的选择，还需要对高度敏感和准确的分子监测技术进行细化和传播。通过逆转录定量聚合酶链反应（qPCR）报告的 BCR-ABL1 信使 RNA 表达的测量，根据国际标准进行报告，已成为 CML 反应评估的主要工具。按照全球治疗指南，实现特定时间依赖性分子里程碑，已被确立为最大化最佳结果并识别有治疗失败风险的患者的关键。在考虑 TFR 的患者中，深度和持续的深度分子反应已成为新的治疗目标。因此，CML 的分子监测对于持续的反应评估变得更加重要，能够识别出 TKI 耐药和药物依从性差的患者，并能够安全有效地尝试 TFR。
