Brittany Klooster, Kaitlin Chatterton, Nazifa Ibrahim, Madison C Bernstein, Alan L Shields, Veleka Allen
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引用次数: 0
Abstract
Background: Chronic rhinosinusitis (inclusive of subtypes with nasal polyps [CRSwNP], without nasal polyps [CRSsNP], and allergic fungal rhinosinusitis [AFRS]) causes inflammation of the nose mucosa and paranasal sinuses. Unfortunately, evidence supporting use of clinical outcome assessments (COAs) in regulated clinical trials to assess key measurement concepts of these conditions is limited.
Objective: To identify key disease-related symptoms and impacts, potential outcomes of interest for new treatments, and COAs available to measure those outcomes among adult and adolescent individuals living with CRSwNP, CRSsNP, and AFRS.
Methods: Literature, clinical trial, and product label reviews were conducted to identify symptoms, impacts, and COAs used to assess CRSwNP, CRSsNP, and AFRS patient experiences in clinical trials. The disease related concepts identified in the literature were mapped to selected COAs to determine conceptual coverage of each COA.
Results: Twenty-five articles, twenty-five clinical trial records, and four product labels were included in the review. Across conditions, nasal obstruction, nasal discharge, and altered smell were the most frequently identified symptoms. The most frequently identified impacts of CRSwNP and CRSsNP were on emotional functioning and sleep, and adopting new behaviors for AFRS. Findings for key symptoms and impacts in adolescents were limited. More than 20 COAs used in these conditions were identified, and 14 COAs (e.g., Sinonasal Outcome Test [SNOT-22]) were evaluated for conceptual coverage of the concepts identified in the literature.
Conclusion: Results specify several symptom and impact outcomes, that if improved, would reflect treatment benefit for patients living with CRSwNP, CRSsNP, and/or AFRS. Several COAs demonstrated coverage of key measurement concepts and warrant further evaluation for use in clinical trials.
背景:慢性鼻窦炎(包括有鼻息肉亚型[CRSwNP]、无鼻息肉亚型[CRSsNP]和变应性真菌性鼻窦炎[AFRS])引起鼻黏膜和鼻窦炎的炎症。不幸的是,支持在规范的临床试验中使用临床结果评估(COAs)来评估这些疾病的关键测量概念的证据有限。目的:确定CRSwNP、crsssnp和AFRS成人和青少年患者的关键疾病相关症状和影响、新疗法的潜在结果,以及可用于测量这些结果的coa。方法:通过文献、临床试验和产品标签审查,确定临床试验中用于评估CRSwNP、crsssnp和AFRS患者体验的症状、影响和coa。将文献中确定的疾病相关概念映射到选定的COA,以确定每个COA的概念覆盖范围。结果:共纳入25篇文献、25项临床试验记录和4个产品标签。在各种情况下,鼻塞、流鼻涕和气味改变是最常见的症状。CRSwNP和crsssnp最常见的影响是对情绪功能和睡眠的影响,以及对AFRS采取新的行为。关于青少年主要症状和影响的发现有限。在这些条件下确定了20多个coa,并评估了14个coa(例如,Sinonasal Outcome Test [SNOT-22])对文献中确定的概念的概念覆盖范围。结论:结果明确了几种症状和影响结果,如果改善,将反映CRSwNP、crsssnp和/或AFRS患者的治疗获益。几个coa证明了关键测量概念的覆盖范围,并保证在临床试验中使用的进一步评估。