Basch Ethan, Rogak Lauren J, Dueck Amylou C
Cancer Outcomes Research Program, Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, North Carolina; Health Outcomes Research Group, Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, New York.
Health Outcomes Research Group, Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, New York.
Clin Ther. 2016 Apr;38(4):821-30. doi: 10.1016/j.clinthera.2016.03.011. Epub 2016 Apr 2.
There is increasing interest to use patient-reported outcome (PRO) measures to evaluate symptomatic adverse events (AEs) in cancer treatment trials. However, there are currently no standard recommended approaches for integrating patient-reported AE measures into trials.
Approaches are identified from previous trials for selecting AEs for solicited patient reporting, administering patient-reported AE measures, and analyzing and reporting results.
Approaches for integrating patient-reported AE measures into cancer trials generally combine current standard methods for clinician-reported AEs and established best practices for using PRO measures. Specific AEs can be selected for a PRO questionnaire based on common and expected reactions in a given trial context, derived from literature review and qualitative/mixed-methods evaluations and should be the same set administered across all arms of a trial. A mechanism for collecting unsolicited patient-reported AEs will also ideally be included. Patients will preferably report at baseline and at the end of active treatment as well as on a frequent standardized schedule during active treatment, such as weekly from home, with a recall period corresponding to the frequency of reporting (eg, past 7 days). Less frequent reporting may be considered after an initial intensive monitoring period for trials of prolonged treatments and during long-term follow-up. Electronic PRO data collection is preferred. Backup data collection for missed PRO reports is advisable to boost response rates. Analysis can use a combination of approaches to AE and PRO data. If a high proportion of patients is experiencing baseline symptoms, systematic subtraction of these from on-study AEs should be considered to improve reporting of symptoms related to treatment. More granular longitudinal analyses of individual symptoms can also be useful.
Methods are evolving for integrating patient-reported symptomatic AEs into cancer trials. These methods are expected to further evolve as more data from trials become available.
在癌症治疗试验中，使用患者报告结局（PRO）指标来评估症状性不良事件（AE）的兴趣日益浓厚。然而，目前尚无将患者报告的AE指标纳入试验的标准推荐方法。
从以往试验中确定选择AE以征求患者报告、实施患者报告的AE指标以及分析和报告结果的方法。
将患者报告的AE指标纳入癌症试验的方法通常结合了当前临床医生报告AE的标准方法和使用PRO指标的既定最佳实践。可以根据给定试验背景下的常见和预期反应，从文献综述以及定性/混合方法评估中选择特定的AE纳入PRO问卷，并且在试验的所有组中应使用相同的一组。理想情况下，还应包括收集非主动报告的患者AE的机制。患者最好在基线、积极治疗结束时以及积极治疗期间按照频繁的标准化时间表进行报告，例如每周在家报告一次，回忆期与报告频率相对应（例如，过去7天）。对于长期治疗试验的初始强化监测期之后以及长期随访期间，可以考虑减少报告频率。首选电子PRO数据收集。建议对遗漏的PRO报告进行备份数据收集以提高回复率。分析可以结合多种AE和PRO数据的方法。如果有很大比例的患者出现基线症状，则应考虑从研究中的AE中系统地减去这些症状以改善与治疗相关症状的报告。对个体症状进行更细致的纵向分析也可能有用。
将患者报告的症状性AE纳入癌症试验的方法正在不断发展。随着更多试验数据的获得，这些方法预计将进一步演变。
