Empirical estimation of disutilities and decision thresholds for composite endpoints

• Type: Article in International Scientific Journal
• Date: 2025

Authors

Sousa-Pinto, B (Author) FMUP; Bognanni, A (Author) Other; Gil-Mata, S (Author) Other; Vieira, RJ (Author) FMUP; Xie, F (Author) Other; Bonovas, S (Author) Other; Piovani, D (Author) Other; Morgano, GP (Author) Other; Ferreira-da-Silva, R (Author) FMUP; Ferreira, A (Author) Other; Cardoso-Fernandes, A (Author) FMUP; Lourenço-Silva, N (Author) Other; Piggott, T (Author) Other; Wiercioch, W (Author) Other; Neumann, I (Author) Other; Bedbrook, A (Author) Other; Yepes-Nuñez, JJ (Author) Other; Klimek, L (Author) Other; Zuberbier, T (Author) Other; Fonseca, JA (Author) Other; Bousquet, J (Author) Other; Schünemann, HJ (Author) Other

Journal

Title: Journal of Clinical Epidemiology

Vol. 179

ISSN: 0895-4356

Publisher: Elsevier

Indexing

ISI Web of Knowledge - 0 Citations

Scopus - 0 Citations

Other information

Authenticus ID: P-017-JKY

DOI: 10.1016/j.jclinepi.2024.111638

Abstract (EN): Objectives: The evaluation of health benefits and harms of an intervention with GRADE Evidence to Decision (EtD) frameworks includes judgments if the effects are trivial,small,moderate,or large.Such judgments ideally require the a priori establishment of decision thresholds (DTs), whose empirical derivation for single outcomes has been previously described. In this article, we provide a methodological approach to estimate DTs for composite endpoints based on disutilities. Study Design and Setting: We generated an approach that involves the computation of pooled disutilities, in which the disutility of each outcome comprised in the composite endpoint is weighted by the respective relative frequency. We also applied a modeling approach based on probability distributions to account for uncertainty in the estimates. We present a practical example of the determination of DTs associated with the development of at least one adverse event following treatment with intranasal medications for rhinitis that we used in the Allergic Rhinitis and its Impact on Asthma guidelines. Results: We provide the methodological steps of a modeling-based approach to compute pooled disutilities and, as a result, DTs for composite endpoints. We have developed a webtool (https://compositedt.med.up.pt/) that allows for a simple implementation of the proposed approach. Applying our approach to a practical example, we concluded that rhinitis nasal medications, compared to placebo, were associated with a trivial harm from adverse events. Conclusion: We propose an approach for estimating DTs for composite endpoints, which may be particularly valuable whenever composite endpoints are used for clinical research, clinical practice, and decision-making. (c) 2024 Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.

Language: English

Type (Professor's evaluation): Scientific

No. of pages: 11