Keeley Tom, Gaw Nina, Ahmed Waseem, Alfonso-Cristancho Rafael, Sousa Ana R, Forde Katie, Sharp Rosie, Whyman Sophie, Gater Adam
Patient Centered Outcomes, GSK, London, UK.
Global Data Generations, GSK, London, UK.
J Patient Rep Outcomes. 2024 Dec 20;8(1):152. doi: 10.1186/s41687-024-00827-4.
PRO measures of symptoms in clinical trials have historically utilized visual and numerical scales but verbal descriptors may make it easier for patients to clearly differentiate between response options. This study assessed content validity and meaningful change in five verbal response scales (VRSs) used to assess chronic rhinosinusitis with nasal polyp (CRSwNP) symptom severity.
This qualitative, semi-structured interview study recruited adults from the US, Germany, and China with confirmed moderate-to-severe CRSwNP. Interviews included a concept elicitation section, where participants were asked about their experience of living with CRSwNP including symptoms and health-related quality-of-life impacts, and a cognitive debriefing section, where participants were debriefed and participant understanding and real-life relevance of the CRSwNP symptom VRS content were assessed. Interview transcripts were qualitatively analyzed using thematic analysis methods.
Among the 24 participants interviewed, the most frequently reported CRSwNP symptoms were nasal obstruction, runny nose, mucus in the throat, loss of smell and facial pain/pressure. Participants demonstrated good understanding of the CRSwNP symptom VRS instructions, items, recall period, and response options. The five CRSwNP symptom VRS items were relevant to the majority of participants' experience of CRSwNP. At the item level, a one-category within-person improvement was the level most frequently reported by participants to be a meaningful change.
The CRSwNP symptom VRSs assess relevant and bothersome symptoms experienced by patients with moderate-to-severe CRSwNP, supporting content validity of this measure. The findings of this study provided preliminary insights into meaningful change in the VRS. Further quantitative assessment of meaningful change is needed, and psychometric evaluation of the CRSwNP symptom VRSs will be required to evaluate their appropriateness for assessment of clinical trial endpoints in patients with CRSwNP.
在临床试验中，用于衡量症状的患者报告结局（PRO）指标历来采用视觉和数字量表，但文字描述可能会让患者更轻松地明确区分不同的反应选项。本研究评估了用于评估伴有鼻息肉的慢性鼻-鼻窦炎（CRSwNP）症状严重程度的五种文字反应量表（VRS）的内容效度和有意义的变化。
这项定性的半结构式访谈研究招募了来自美国、德国和中国的确诊为中重度CRSwNP的成年人。访谈包括一个概念引出部分，在此部分中，参与者被问及他们患CRSwNP的生活经历，包括症状以及对健康相关生活质量的影响；还有一个认知反馈部分，在此部分中，对参与者进行反馈，并评估他们对CRSwNP症状VRS内容的理解以及与现实生活的相关性。使用主题分析方法对访谈记录进行定性分析。
在接受访谈的24名参与者中，最常报告的CRSwNP症状是鼻塞、流涕、咽喉部黏液、嗅觉丧失和面部疼痛/压迫感。参与者对CRSwNP症状VRS的说明、条目、回忆期和反应选项表现出良好的理解。五个CRSwNP症状VRS条目与大多数参与者的CRSwNP经历相关。在条目层面，参与者最常报告的有意义变化水平是一个类别内的个人改善。
CRSwNP症状VRS评估了中重度CRSwNP患者经历的相关且困扰的症状，支持了该指标的内容效度。本研究结果为VRS中有意义的变化提供了初步见解。需要对有意义的变化进行进一步的定量评估，并且需要对CRSwNP症状VRS进行心理测量学评估，以评估它们在评估CRSwNP患者临床试验终点方面的适用性。
