Bryant Ashley Leak, Drier Sarah W, Lee Sejin, Bennett Antonia V
School of Nursing, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Cancer Outcomes Research Program, Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Cancer Outcomes Research Program, Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Leuk Res. 2018 Jul;70:106-116. doi: 10.1016/j.leukres.2018.06.006. Epub 2018 Jun 7.
The purpose of this systematic literature review was to identify clinical trials of MDS and AML that included patient-reported outcome (PRO) instruments, and to summarize the symptom and other health related quality of life (HRQOL) concepts most frequently assessed and the PRO instruments that were used. Sixteen manuscripts describing 14 distinct trials met all criteria (i.e., phase 2 or 3 clinical trial for MDS or AML which included PRO assessment) and were published between 1996-2017. In trials evaluating anemia, PRO scores showed significant improvement in relevant domains (e.g. fatigue, function) among patients identified as responders. In trials evaluating the impact of anti-cancer therapies, improvements the baseline to end of treatment were observed in physical functioning and HRQOL, however the rates of missing data in many of the trials was high or unreported. PRO instruments have the ability to capture changes over time in patients' function and well-being, and PRO instruments and guidance documents are available to support the assessment of HRQOL in AML/MDS clinical trials.
本系统文献综述的目的是识别包含患者报告结局（PRO）工具的骨髓增生异常综合征（MDS）和急性髓系白血病（AML）的临床试验，并总结最常评估的症状及其他与健康相关的生活质量（HRQOL）概念，以及所使用的PRO工具。16篇描述14项不同试验的手稿符合所有标准（即MDS或AML的2期或3期临床试验，其中包括PRO评估），并于1996年至2017年期间发表。在评估贫血的试验中，PRO评分显示在被确定为有反应的患者中，相关领域（如疲劳、功能）有显著改善。在评估抗癌治疗影响的试验中，观察到从基线到治疗结束时身体功能和HRQOL有所改善，然而许多试验中的数据缺失率很高或未报告。PRO工具能够捕捉患者功能和幸福感随时间的变化，并且有PRO工具和指导文件可用于支持AML/MDS临床试验中的HRQOL评估。
