Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases, Rome, Italy.
L. and A. Seràgnoli Institute of Hematology, Department of Experimental, Diagnostic, and Specialty Medicine, S. Orsola-Malpighi University Hospital, University of Bologna, Bologna, Italy.
Cancer. 2018 May 15;124(10):2228-2237. doi: 10.1002/cncr.31323. Epub 2018 Mar 2.
Although a wealth of efficacy and safety data is available for many tyrosine kinase inhibitors used in chronic myeloid leukemia (CML), there is a dearth of information on their impact on patients' health-related quality of life (HRQOL). The primary objective of this study was to evaluate HRQOL and fatigue outcomes in patients with CML receiving first-line therapy with nilotinib.
This was a multicenter, prospective study enrolling 130 patients with chronic-phase CML. HRQOL and fatigue were evaluated with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) and its validated Fatigue module at the baseline and then at 3, 6, 12, 18, and 24 months. The primary prespecified HRQOL endpoints defined in the study protocol for longitudinal analysis were the Physical Functioning, Social Functioning, Role Functioning, and Fatigue scales. The remaining scales were investigated on an exploratory basis.
The rate of baseline compliance with the HRQOL assessment was 95.4% (124 of 130), and the rate of overall compliance with HRQOL forms was 91%. Among the 4 prespecified primary HRQOL endpoints, statistically significant improvements over time were found for Physical Functioning (P = .013), Role Functioning (P = .004), and Fatigue (P < .001). Clinically meaningful improvements were found already 3 months after the treatment start. The baseline patient self-reported fatigue severity was an independent predictive factor for the achievement of a major molecular response with an odds ratio of 0.960 (95% confidence interval, 0.934-0.988; P = .005).
For most patients, HRQOL improvements with nilotinib occur during the early phase of therapy and are maintained over time. Also, a more systematic HRQOL evaluation during the diagnostic workup of CML may help to predict clinical outcomes. Cancer 2018;124:2228-37. © 2018 American Cancer Society.
虽然有大量关于用于慢性髓性白血病（CML）的多种酪氨酸激酶抑制剂的疗效和安全性数据，但关于其对患者健康相关生活质量（HRQOL）影响的信息却很少。本研究的主要目的是评估接受一线治疗的 CML 患者的 HRQOL 和疲劳结果，这些患者使用的一线治疗药物为尼洛替尼。
这是一项多中心、前瞻性研究，共纳入 130 例慢性期 CML 患者。采用欧洲癌症研究与治疗组织生活质量问卷核心 30 项（EORTC QLQ-C30）及其验证的疲劳模块在基线时，然后在 3、6、12、18 和 24 个月时评估 HRQOL 和疲劳情况。研究方案中规定的用于纵向分析的主要预设 HRQOL 终点是身体功能、社会功能、角色功能和疲劳量表。其余量表则进行了探索性分析。
基线时 HRQOL 评估的依从率为 95.4%（130 例中有 124 例），HRQOL 表格的总体依从率为 91%。在 4 个预设的主要 HRQOL 终点中，随着时间的推移，身体功能（P =.013）、角色功能（P =.004）和疲劳（P <.001）均有显著改善。治疗开始后 3 个月就发现了有临床意义的改善。基线时患者报告的疲劳严重程度是实现主要分子反应的独立预测因素，优势比为 0.960（95%置信区间，0.934-0.988；P =.005）。
对于大多数患者来说，尼洛替尼治疗期间 HRQOL 的改善发生在早期阶段，并随着时间的推移而保持。此外，在 CML 的诊断过程中更系统地进行 HRQOL 评估可能有助于预测临床结果。癌症 2018；124：2228-37。©2018 美国癌症协会。
