An Update on Patient-Reported Outcomes in Asthma.

Topic Importance: Patient-reported outcomes (PROs) are information provided by patients on their condition, function, well-being, or experience. Instruments to quantify PROs, called patient-reported outcome measures (PROMs), allow standardized assessment of a unique dimension of health that cannot be measured physically. Herein, we discuss how to appraise PROMs critically and provide an update on their use in asthma clinical practice and research.
Review Findings: Asthma-specific PROMs have been developed to measure a wide array of disease characteristics, including symptoms, medication use, exacerbations, and impairments to emotional and physical function. Some PROMs also include spirometry or expand questions to overlap with rhinitis symptoms. Use of PROMs to understand asthma control is included in management guidelines, yet real-world evidence of their effectiveness in improving asthma care remains limited. These instruments may be less accurate in characterizing patients with poorly controlled asthma and have modest correlation with exacerbation risk. Two new PROMs are highlighted, the Asthma Impairment and Risk Questionnaire as an instrument to assess asthma control that incorporates domains related to exacerbation risk and impairment, and the CompEx as a composite of daily diary reporting combined with exacerbation events as an early efficacy signal for interventional trials.
Summary: PROMs are fundamental to asthma assessment. Novel instruments may improve the detection of patients at risk for poor outcomes and shorten the drug discovery pipeline. However, urgent research is needed to understand their practical utility in clinical settings.
Competing Interests: Financial/Nonfinancial Disclosures The authors have reported to CHEST the following: Z. D. received speaker or consultant honoraria and/or served on advisory boards at: Antabio, Arcede, Foresee Pharmaceuticals, GlaxoSmithKline, Hippo-Dx, QPS-Netherlands, and Sanofi-Genzyme-Regeneron, all outside the submitted work. During the last 3 years of Z. D.’s assignment as Research Director Respiratory and Allergy, QPS-Netherlands received a European grant from ERA4TB and funding from Foresee Pharmaceuticals for early clinical studies. She served/serves as associate editor for Allergy and Respiratory Medicine and is past chair of the asthma expert panel at EUFOREA (2000-2023) and past chair of the EAACI Asthma Section (2017-2019). N. A. H. has received honoraria for serving as consultant/advisor from Sanofi/Regeneron, Genentech, Astra Zeneca, Amgen, GSK, and Boehringer Ingelheim. His institution receives research grant support on his behalf from Genentech, Astra Zeneca, GSK, and Sanofi. None declared (T. D. W.).
(Copyright © 2024. Published by Elsevier Inc.)