Hagelstein V, Ortland I, Wilmer A, Mitchell S A, Jaehde U
Institute of Pharmacy, Clinical Pharmacy, University of Bonn, Bonn, Germany.
Division of Cancer Control and Population Sciences, Outcomes Research Branch, National Cancer Institute, Rockville, USA.
Ann Oncol. 2016 Dec;27(12):2294-2299. doi: 10.1093/annonc/mdw422. Epub 2016 Sep 28.
Integrating the patient's perspective has become an increasingly important component of adverse event reporting. The National Cancer Institute has developed a Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™). This instrument has been translated into German and linguistically validated; however, its quantitative measurement properties have not been evaluated.
A German language survey that included 31 PRO-CTCAE items, as well as the EORTC QLQ-C30 and the Oral Mucositis Daily Questionnaire (OMDQ), was distributed at 10 cancer treatment settings in Germany and Austria. Item quality was assessed by analysis of acceptability and comprehensibility. Reliability was evaluated by using Cronbach's' alpha and validity by principal components analysis (PCA), multitrait-multimethod matrix (MTMM) and known groups validity techniques.
Of 660 surveys distributed to the study centres, 271 were returned (return rate 41%), and data from 262 were available for analysis. Participants' median age was 59.7 years, and 69.5% of the patients were female. Analysis of item quality supported the comprehensibility of the 31 PRO-CTCAE items. Reliability was very good; Cronbach's' alpha correlation coefficients were >0.9 for almost all item clusters. Construct validity of the PRO-CTCAE core item set was shown by identifying 10 conceptually meaningful item clusters via PCA. Moreover, construct validity was confirmed by the MTMM: monotrait-heteromethod comparison showed 100% high correlation, whereas heterotrait-monomethod comparison indicated 0% high correlation. Known groups validity was supported; PRO-CTCAE scores were significantly lower for those with impaired versus preserved health-related quality of life.
A set of 31 items drawn from the German PRO-CTCAE item library demonstrated favourable measurement properties. These findings add to the body of evidence that PRO-CTCAE provides a rigorous method to capture patient self-reports of symptomatic toxicity for use in cancer clinical trials.
将患者的观点纳入不良事件报告已成为一个日益重要的组成部分。美国国立癌症研究所已开发出不良事件通用术语标准的患者报告结局版本（PRO-CTCAE™）。该工具已被翻译成德语并进行了语言验证；然而，其定量测量属性尚未得到评估。
一项德语调查问卷在德国和奥地利的10个癌症治疗机构发放，问卷包含31个PRO-CTCAE项目，以及欧洲癌症研究与治疗组织核心问卷（EORTC QLQ-C30）和口腔黏膜炎每日问卷（OMDQ）。通过分析可接受性和可理解性来评估项目质量。使用克朗巴赫α系数评估信度，通过主成分分析（PCA）、多特质多方法矩阵（MTMM）和已知群体效度技术评估效度。
在分发给研究中心的660份调查问卷中，回收了271份（回收率41%），262份的数据可供分析。参与者的中位年龄为59.7岁，69.5%的患者为女性。项目质量分析支持了31个PRO-CTCAE项目的可理解性。信度非常好；几乎所有项目组的克朗巴赫α相关系数均>0.9。通过PCA识别出10个具有概念意义的项目组，表明PRO-CTCAE核心项目集具有结构效度。此外，MTMM证实了结构效度：单特质-异方法比较显示高度相关性为100%，而异特质-单方法比较显示高度相关性为0%。已知群体效度得到支持；健康相关生活质量受损者的PRO-CTCAE得分显著低于健康相关生活质量未受损者。
从德语PRO-CTCAE项目库中选取的一组31个项目表现出良好的测量属性。这些发现进一步证明了PRO-CTCAE为在癌症临床试验中获取患者对症状性毒性的自我报告提供了一种严谨的方法。
