Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

European respiratory review

article

Meena Kalluri¹  Elisabetta Renzoni²  Sebastiano Torrisi³  Mariarosaria Calvello⁴  Alessandro Libra⁵  Mauro Pavone³  Francesco Bonella⁶  Vincent Cottin⁷  Claudia Valenzuela⁹  Marlies Wijsenbeek^1,10  Elisabeth Bendstrup^1,11  Fabrizio Luppi^1,12  Ada Vancheri⁵  Carlo Vancheri³  Elisabetta Balestro^1,13  Francesco Varone⁴  Nesrin Mogulkoc^1,14  Giulia Cacopardo^1,15  Elena Bargagli^1,16 

[1] Division of Pulmonary Medicine, University of Alberta;Interstitial Lung Disease Unit, Royal Brompton Hospital, Imperial College;Dept of Clinical and Experimental Medicine, Regional Referral Centre for Rare Lung Diseases, University - Hospital “Policlinico G. Rodolico – San Marco”, University of Catania;UOC Pneumologia, Fondazione Policlinico A. Gemelli IRCCS;Regional Referral Center for Rare Lung Diseases, University - Hospital “Policlinico G. Rodolico – San Marco”;Centre for Interstitial and Rare Lung Disease, Ruhrlandklinik University Hospital, University of Duisburg-Essen;Dept of Respiratory Medicine, National Reference Coordinating Center for Rare Pulmonary Diseases, Louis Pradel Hospital;University of Lyon;Hospital Universitario de la Princesa, Universidad Autonoma Madrid;Centre of excellence, University Medical Centre Rotterdam;Centre for Rare Lung Diseases, Aarhus University Hospital;Respiratory Diseases Unit, University of Milano-Bicocca. “S. Gerardo” Hospital;Vascular Sciences and Public Health, University of Padova;Ege University Hospiral;UOSD UTIR, A.R.N.A.S. Ospedali Civico Di Cristina Benfratelli;Respiratory Diseases Unit, Siena University

DOI  :  10.1183/16000617.0026-2021

学科分类：工程和技术（综合）

来源: European Respiratory Society

【 摘 要 】

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

【 授权许可】

CC BY-NC