Department of Otolaryngology Head and Neck Surgery, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Faculty of Medicine, Health and Human Sciences, Macquarie University, Sydney, Australia.
Rhinology. 2024 Aug 31;62(34):1-37. doi: 10.4193/Rhin24.090.
With the recent proliferation of novel therapeutics for chronic rhinosinusitis with nasal polyps (CRSwNP), there is an immediate need for comprehensive means to assess CRSwNP disease status as well as to determine treatment efficacy. Outcome measures exist in different forms. Patient-reported outcome measures (PROMs) allow patients to provide direct input about their condition that is not possible to obtain in any other way. Common constructs that are measured using PROMs include quality of life or the burden of disease manifestations (e.g., symptom severity). Outcomes may also include the results of objective diagnostic testing/measurement of clinical signs or measured using psychophysical tests. Biomarkers represent an emerging class of outcome measures for CRSwNP and are chosen to directly reflect the active pathophysiologic processes of CRSwNP in the peripheral blood, sinus/polyp tissues, and sinonasal mucus.
Narrative review of the literature, identifying and describing outcome measures that may be used in the evaluation of CRSwNP and for assessment of treatment responses.
In this review, we identify many different outcome measures for CRSwNP that fall under the categories of PROM, objective test, psychophysical test or biomarker. We describe the history of each - including seminal studies - and demonstrate the formal validation, psychometric performance, and limitations of each.
PROMs, objective tests, psychophysical tests and biomarkers represent different classes of outcome measures that are complementary means of assessing CRSwNP disease status and treatment efficacy. The choice or interpretation of a CRSwNP outcome measure should be undertaken with full knowledge of its formal validation, psychometric performance, and limitations.
随着新型治疗慢性鼻-鼻窦炎伴鼻息肉（CRSwNP）药物的不断涌现，我们迫切需要全面的方法来评估 CRSwNP 的疾病状态，并确定治疗效果。现已有多种评估方法。患者报告结局（PROM）可让患者直接提供其病情的信息，而这些信息无法通过其他方式获得。常用的 PROM 测量指标包括生活质量或疾病表现的负担（如症状严重程度）。结果也可能包括客观诊断测试/临床体征的测量结果，或使用心理物理测试进行测量。生物标志物是 CRSwNP 新兴的一类评估指标，它是为了直接反映 CRSwNP 外周血、鼻窦/息肉组织和鼻黏膜的活跃病理生理过程而选择的。
对文献进行叙述性综述，确定并描述可用于评估 CRSwNP 和评估治疗反应的评估指标。
在本综述中，我们确定了许多不同的 CRSwNP 评估指标，这些指标属于 PROM、客观测试、心理物理测试或生物标志物。我们描述了每种评估指标的历史，包括开创性研究，并展示了它们的正式验证、心理测量性能和局限性。
PROM、客观测试、心理物理测试和生物标志物代表不同类别的评估指标，它们是评估 CRSwNP 疾病状态和治疗效果的互补手段。选择或解释 CRSwNP 评估指标时，应充分了解其正式验证、心理测量性能和局限性。
