McCarrier Kelly P, Hassan Mariam, Hodgkins Paul, Suthoff Ellison, McGarry Lisa J, Martin Mona L
Pharmerit International, 4350 East-West Highway, Suite 1100, Bethesda, MD, 20814, USA.
Insmed Pharmaceuticals, Bridgewater, NJ, USA.
J Patient Rep Outcomes. 2020 May 13;4(1):36. doi: 10.1186/s41687-020-00199-5.
Patients with cystic fibrosis (CF) experience significant disease burden, including progressive pulmonary decline and reduced survival. This multicenter qualitative study was conducted to develop a new patient-reported outcome (PRO) measure to assess the impact of CF on patients' quality of life: the Cystic Fibrosis Impact Questionnaire (CF-IQ). Semi-structured qualitative concept elicitation (CE) interviews with patients and caregivers documented CF-related symptoms, impacts, and treatment experiences. Coded interview data were considered alongside existing PROs, published literature, and expert opinion to develop an initial scale. Three rounds of cognitive interviews evaluated respondent comprehension and facilitated refinement of the CF-IQ.
Adult (N = 20) and pediatric (N = 22) patients with CF and their parents/caregivers (N = 22) completed CE interviews at 7 US clinics. The sample included patients aged 6-58 years, 57% females, and represented a broad range of disease severity (forced expiratory volume in 1 s range: 22%-127% predicted). Interviews identified 59 unique CF-related impact concepts in domains, including activity limitations (physical, social, leisure), functional limitations (school, work), vulnerability/lack of control, emotional impact, treatment burden, and future outlook. Concept saturation was achieved, and a draft questionnaire was developed. Findings from the cognitive interviews (n = 18) confirmed that instructions, items, and response scales were relevant and clear, and interpreted as intended by patients.
The CF-IQ is a 40-item novel PRO scale assessing a comprehensive set of patient-relevant concepts to characterize the multifaceted nature of CF. Qualitative interview data support the content validity of the CF-IQ, which is currently undergoing additional psychometric evaluation in patients with CF.
囊性纤维化（CF）患者承受着巨大的疾病负担，包括进行性肺功能下降和生存率降低。本多中心定性研究旨在开发一种新的患者报告结局（PRO）指标，以评估CF对患者生活质量的影响：囊性纤维化影响问卷（CF-IQ）。对患者及其护理人员进行半结构化定性概念激发（CE）访谈，记录与CF相关的症状、影响和治疗经历。将编码后的访谈数据与现有的PRO指标、已发表的文献以及专家意见相结合，以制定初始量表。三轮认知访谈评估了受访者的理解情况，并促进了CF-IQ的完善。
20名成年CF患者、22名儿科CF患者及其22名父母/护理人员在美国的7家诊所完成了CE访谈。样本包括6至58岁的患者，57%为女性，代表了广泛的疾病严重程度（1秒用力呼气量范围：预测值的22%至127%）。访谈在多个领域确定了与CF相关的59个独特影响概念，包括活动限制（身体、社交、休闲）、功能限制（学校、工作）、易损性/缺乏控制、情绪影响、治疗负担和未来展望。达到了概念饱和，并制定了问卷草稿。认知访谈（n = 18）的结果证实，指导语、条目和反应量表相关且清晰，患者的理解与预期一致。
CF-IQ是一个包含40个条目的新型PRO量表，评估了一组与患者相关的综合概念，以描述CF的多面性。定性访谈数据支持CF-IQ的内容效度，该量表目前正在CF患者中进行进一步的心理测量学评估。
