非小细胞肺癌症状评估问卷：一种新型患者报告症状测量方法的心理测量性能和监管资质
Non-Small Cell Lung Cancer Symptom Assessment Questionnaire: Psychometric Performance and Regulatory Qualification of a Novel Patient-Reported Symptom Measure.
作者信息
Bushnell Donald M, Atkinson Thomas M, McCarrier Kelly P, Liepa Astra M, DeBusk Kendra P, Coons Stephen Joel
机构信息
Patient-Centered Research, Evidera, Seattle, Washington.
Memorial Sloan Kettering Cancer Center, Patient-Reported Outcomes, Community-Engagement, and Language Core Facility, New York, New York.
出版信息
Curr Ther Res Clin Exp. 2021 Aug 26;95:100642. doi: 10.1016/j.curtheres.2021.100642. eCollection 2021.
BACKGROUND
The Non-Small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ) was developed to incorporate the patient's perspective into evaluation of clinical benefit in advanced non-small cell lung cancer trials and meet regulatory expectations for doing so. Qualitative evidence supported 7 items covering 5 symptom concepts.
OBJECTIVE
This study evaluated measurement properties of the NSCLC-SAQ's items, overall scale, and total score.
METHODS
In this observational cross-sectional study, a purposive sample of patients with clinician-diagnosed advanced non-small cell lung cancer, initiating or undergoing treatment, provided sociodemographic information and completed the NSCLC-SAQ, National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy Lung Symptom Index (FLSI-17), and a Patient Global Impression of Severity item. Rasch analyses, factor analyses, and assessments of construct validity and reliability were completed.
RESULTS
The 152 participants had a mean age of 64 years, 57% were women, and 87% where White. The majority were Stage IV (83%), 51% had an Eastern Cooperative Oncology Group performance status of 1 (32% performance status 0 and 17% performance status 2), and 33% were treatment naïve. Rasch analyses showed ordered thresholds for response options. Factor analyses demonstrated that items could be combined for a total score. Internal consistency (Cronbach α = 0.78) and test-retest reliability (intraclass correlation coefficient = 0.87) were quite satisfactory. NSCLC-SAQ total score correlation was 0.83 with the National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy Lung Symptom Index-17. The NSCLC-SAQ was able to differentiate between symptom severity levels and performance status (both values < .001).
CONCLUSIONS
The NSCLC-SAQ generated highly reliable scores with substantial evidence of construct validity. The Food and Drug Administration's qualification supports the NSCLC-SAQ as a measure of symptoms in drug development. Further evaluation is needed on its longitudinal measurement properties and interepretation of meaningful within-patient score change. (Curr Ther Res Clin Exp. 2021; 82:XXX-XXX).
背景
非小细胞肺癌症状评估问卷（NSCLC-SAQ）的开发旨在将患者的观点纳入晚期非小细胞肺癌试验的临床获益评估中，并满足监管方面的相关期望。定性证据支持涵盖5个症状概念的7个条目。
目的
本研究评估了NSCLC-SAQ各条目、整体量表及总分的测量属性。
方法
在这项观察性横断面研究中，选取了有临床诊断的晚期非小细胞肺癌且正在开始或接受治疗的患者作为目标样本，收集其社会人口学信息，并让他们完成NSCLC-SAQ、美国国立综合癌症网络/癌症治疗功能评估肺癌症状指数（FLSI-17）以及一项患者总体严重程度印象条目。完成了拉施分析、因子分析以及结构效度和信度评估。
结果
152名参与者的平均年龄为64岁，57%为女性，87%为白人。大多数为IV期（83%），51%的东部肿瘤协作组体能状态为1（32%体能状态为0，17%体能状态为2），33%为初治患者。拉施分析显示了反应选项的有序阈值。因子分析表明各条目可合并计算总分。内部一致性（克朗巴哈α系数=0.78）和重测信度（组内相关系数=0.87）相当令人满意。NSCLC-SAQ总分与美国国立综合癌症网络/癌症治疗功能评估肺癌症状指数-17的相关性为0.83。NSCLC-SAQ能够区分症状严重程度水平和体能状态（两者P值均<0.001）。
结论
NSCLC-SAQ产生了高度可靠的分数，并有大量结构效度证据。美国食品药品监督管理局的资质认定支持NSCLC-SAQ作为药物研发中症状的一项测量指标。需要对其纵向测量属性以及患者内有意义的分数变化的解读进行进一步评估。（《当前治疗研究与临床实验》。2021年；82:XXX - XXX）
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