University of Leeds, Leeds, UK.
University of Bristol, Bristol, UK.
Lancet. 2015 Feb 26;385 Suppl 1:S38. doi: 10.1016/S0140-6736(15)60353-1.
In the USA, more than 7200 new cases of anal cancer were diagnosed in 2014 with incidence rising. Concurrent chemoradiotherapy improves cancer-related outcomes but has led to an increase in acute and late adverse events. Patient-reported outcomes (PRO) are increasingly included in trials as a surrogate measure for reporting of late adverse events. This study aimed to select the most effective PRO to use in clinical research and practice for anal cancer since no questionnaire specific for anal cancer has been developed.
A mixed methods approached was used. A systematic review estimated the frequency of use of PROs and clinician reporting instruments used in anal cancer trials including radiation treatment. Health professional semi-structured interviews explored preferred questionnaires and revealed missing items; analysis was done using the framework approach. Two symptom-based, validated questionnaires were selected and assessed by means of inductive content analysis to highlight discrepancies relevant to anal cancer.
34 relevant studies reported on radiotherapy adverse events. For PROs, EORTC QLQ-C30 (n=5) and EORTC QLQ-CR38 (n=3) were used most frequently. RTOG/EORTC (n=17) and CTCAE (n=15) criteria were most commonly used for clinician reporting. EORTC QLQ-C30 and EORTC QLQ-CR38, and National Cancer Institute's (NCI) PRO-CTCAE were selected for further analysis. No consensus for questionnaire content or design was found through health professional interviews (n=8). Domains and codes relevant to anal cancer treatment were selected from interviews to inform questionnaire analysis. 27 domains and 60 codes were found in EORTC questionnaires, and 21 domains and 44 codes in NCI PRO-CTCAE. Four domains and 16 codes were not covered by EORTC systems including radiation skin reaction. Six domains and 45 codes were not covered by NCI PRO-CTCAE including vaginal stenosis and bowel urgency.
This study shows that there are gaps in the questionnaires commonly used to record adverse events using PROs for anal cancer. Expert opinion is valuable in highlighting relevant missing items but provides no consensus on design and wording preferences. For use in longitudinal follow-up of patients with anal cancer treated with (chemo)radiation, the EORTC-QLQ system has the fewest missing symptom items for use as a validated PRO in clinical research and practice.
This abstract presents independent research funded by the National Institute for Health Research (NIHR).
2014 年，美国诊断出超过 7200 例新的肛门癌病例，发病率呈上升趋势。同期放化疗可改善癌症相关结局，但会导致急性和晚期不良事件的增加。患者报告的结局（PRO）越来越多地被纳入试验中，作为报告晚期不良事件的替代指标。本研究旨在选择最有效的 PRO 用于肛门癌的临床研究和实践，因为目前尚无专门针对肛门癌的问卷。
采用混合方法。系统评价估计了肛门癌试验中使用的 PRO 和临床医生报告工具的使用频率，包括放射治疗。对卫生专业人员进行半结构化访谈，探讨首选问卷，并揭示缺失项目；使用框架方法进行分析。选择了两种基于症状的、经过验证的问卷，并通过归纳内容分析进行评估，以突出与肛门癌相关的差异。
34 项相关研究报告了放疗不良事件。对于 PRO，EORTC QLQ-C30（n=5）和 EORTC QLQ-CR38（n=3）使用最频繁。RTOG/EORTC（n=17）和 CTCAE（n=15）标准最常用于临床医生报告。选择 EORTC QLQ-C30 和 EORTC QLQ-CR38 以及美国国家癌症研究所（NCI）的 PRO-CTCAE 进行进一步分析。通过卫生专业人员访谈（n=8）未发现问卷内容或设计的共识。从访谈中选择与肛门癌治疗相关的领域和代码，以告知问卷分析。在 EORTC 问卷中发现了 27 个领域和 60 个代码，在 NCI PRO-CTCAE 中发现了 21 个领域和 44 个代码。EORTC 系统未涵盖包括放射性皮肤反应在内的 4 个领域和 16 个代码。NCI PRO-CTCAE 未涵盖包括阴道狭窄和肠急迫在内的 6 个领域和 45 个代码。
本研究表明，用于记录肛门癌患者（放化疗）后不良事件的 PRO 中，常用的问卷存在差距。专家意见对于突出相关缺失项目很有价值，但在设计和措辞偏好方面没有达成共识。对于接受（化疗）放疗的肛门癌患者的纵向随访，EORTC-QLQ 系统在作为临床研究和实践中验证的 PRO 使用时，具有最少的缺失症状项目。
本摘要介绍了由英国国家卫生研究院（NIHR）资助的独立研究。
