Kawaguchi Takashi, Azuma Kanako, Sano Motohiko, Kim Soan, Kawahara Yosuke, Sano Yoko, Shimodaira Tomohide, Ishibashi Keiichiro, Miyaji Tempei, Basch Ethan, Yamaguchi Takuhiro
1Department of Practical Pharmacy, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences, 1432-1, Horinouchi, Hachioji, Tokyo, Japan.
2Department of Pharmacy, Tokyo Medical University Hospital, 6-7-1, Nishishinjuku, Shinjuku-ku, Tokyo, Japan.
J Patient Rep Outcomes. 2017;2(1):2. doi: 10.1186/s41687-017-0022-5. Epub 2018 Jan 5.
The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) was developed by the National Cancer Institute as an adverse event assessment system to evaluate patients' symptoms, which tend to be underestimated in cancer clinical trials. The aim of this study was to assess the psychometric properties of the Japanese version of the PRO-CTCAE and the degree of adverse event assessment discordance between clinicians and patients.
A total of 187 cancer patients receiving systemic therapy were enrolled. Reproducibility, criterion validity, and responsiveness of the Japanese version of PROCTCAE were assessed. The EORTC QLQ-C30 was used as an external anchor. Discordance of assessment of adverse events between clinician and patients were also assessed using the CTCAE and PRO-CTCAE.
A total of 187 participants (187 for criterion validity, 80 for reproducibility, and 100 for responsiveness), were analyzed (Mage = 62.4 years). All patients responded to at least one symptom item (M = 16). The mean (SD) intra-class correlation coefficients of overall reproducibility for the Japanese PRO-CTCAE was 0.63 (0.02). The correlation coefficient for the corresponding items in the EORTC QLQ-C30 and the Japanese PRO-CTCAE was high (Pearson = 0.56-0.76). The analysis of responsiveness revealed significant dose-response trends (Jonckheere-Terpstra test, ps < 0.001). Depending on the adverse events, a discrepancy was observed in evaluation between the clinician and patient.
These results revealed that there is underestimation in the assessment of adverse events in Japan, and that the Japanese version of the PRO-CTCAE had acceptable reliability and validity for common and clinically important symptoms.
患者报告的不良事件通用术语标准（PRO-CTCAE）由美国国立癌症研究所开发，作为一种不良事件评估系统，用于评估患者的症状，而这些症状在癌症临床试验中往往被低估。本研究的目的是评估PRO-CTCAE日语版的心理测量特性以及临床医生与患者之间不良事件评估的不一致程度。
共纳入187例接受全身治疗的癌症患者。评估了PRO-CTCAE日语版的可重复性、标准效度和反应性。欧洲癌症研究与治疗组织生活质量核心问卷（EORTC QLQ-C30）用作外部对照。还使用CTCAE和PRO-CTCAE评估了临床医生与患者之间不良事件评估的不一致性。
共分析了187名参与者（标准效度分析187例，可重复性分析80例，反应性分析100例）（平均年龄=62.4岁）。所有患者至少对一项症状条目做出了回答（平均回答数=16项）。PRO-CTCAE日语版总体可重复性的组内相关系数均值（标准差）为0.63（0.02）。EORTC QLQ-C30与PRO-CTCAE日语版中相应条目的相关系数较高（Pearson相关系数=0.56-0.76）。反应性分析显示出显著的剂量反应趋势（Jonckheere-Terpstra检验，P值<0.001）。根据不良事件的不同，临床医生与患者之间的评估存在差异。
这些结果表明，在日本不良事件评估中存在低估现象，且PRO-CTCAE日语版对于常见且具有临床重要性的症状具有可接受的信度和效度。
