Department Hematology, Hull University Teaching Hospitals NHS Trust, Hull, UK.
Expert Rev Hematol. 2024 Jul;17(7):279-285. doi: 10.1080/17474086.2024.2366534. Epub 2024 Jun 13.
Traditionally, the success of chronic lymphocytic leukemia (CLL) treatment has been primarily assessed based on clinical outcomes, such as disease response, progression-free survival (PFS), and overall survival (OS). However, the evolution of treatment approaches recognizes the importance of a patient-centered perspective that includes factors directly affecting patients' quality of life and overall well-being.
Studies addressing the impact of targeted agents on improving either OS or other endpoint surrogates were selected using PubMed and MEDLINE platforms. Our search also included studies that considered patient-centric endpoints such as health-related quality of life and patient-reported outcomes (PROs).
The changing landscape of CLL treatment underscores the importance of continually exploring various endpoints to thoroughly define treatment success. Beyond conventional metrics such as OS and surrogate endpoints, namely, PFS, time to next treatment (TTNT), and measurable residual disease (MRD) assessment, it becomes crucial to integrate enhanced comorbidity evaluations and patient-centered viewpoints into a CLL success roadmap.Subsequent investigations ought to concentrate on enhancing current surrogate endpoints, discerning their contextual significance, and exploring innovative indicators of treatment efficacy and safety. Given the dynamic nature of CLL and the heterogeneity among patient groups, personalized strategies are essential, taking into account individual traits and patient preferences.
传统上,慢性淋巴细胞白血病 (CLL) 治疗的成功主要基于临床结果进行评估,例如疾病反应、无进展生存期 (PFS) 和总生存期 (OS)。然而,治疗方法的发展认识到以患者为中心的观点的重要性,其中包括直接影响患者生活质量和整体幸福感的因素。
使用 PubMed 和 MEDLINE 平台选择了评估靶向药物对改善 OS 或其他终点替代指标影响的研究。我们的搜索还包括考虑患者为中心的终点的研究,例如健康相关生活质量和患者报告的结果 (PROs)。
CLL 治疗领域的变化强调了不断探索各种终点以全面定义治疗成功的重要性。除了 OS 和替代终点(即 PFS、下一次治疗时间 (TTNT) 和可测量残留疾病 (MRD) 评估)等传统指标外,评估合并症和纳入患者为中心的观点对于制定 CLL 成功路线图至关重要。后续研究应集中于增强当前替代终点,辨别其背景意义,并探索治疗效果和安全性的创新指标。鉴于 CLL 的动态性质和患者群体之间的异质性,需要个体化策略,考虑个体特征和患者偏好。