Abstract
Patient-reported outcome instruments provide valuable insight into disease-related morbidity known only to the patient and complement more objective outcome tools in the clinical trial setting. They are of particular importance in systemic sclerosis owing to the challenges around defining disease activity, the episodic nature of many disease-specific manifestations and the paucity of validated objective surrogate outcome measures for use in clinical trials. Early clinical trials of systemic sclerosis often incorporated legacy patient-reported outcome instruments, but the last 20 years has witnessed the emergence of several scleroderma-specific instruments that are now being routinely used alongside other outcomes in systemic sclerosis clinical trials. More recently, the value of patient-reported outcomes has been highlighted by their prominence in the American College of Rheumatology Combined Response Index for Systemic Sclerosis that has been utilized as the primary endpoint of recent clinical trials of early diffuse systemic sclerosis. This review considers the role and performance of the various patient-reported outcome instruments utilized in systemic sclerosis clinical trials, the current positioning of patient-reported outcome instruments within clinical trial endpoint models across the range of systemic sclerosis disease manifestations and, where applicable, we shall highlight areas for future research.
Original language | English |
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Pages (from-to) | 90-102 |
Journal | Journal of Scleroderma and Related Disorders |
Volume | 5 |
Issue number | 2 |
Early online date | 25 Nov 2019 |
DOIs | |
Publication status | Published - 1 Jun 2020 |
Funding
https://orcid.org/0000-0002-2793-2364 Pauling John D 1 2 Caetano Joana 3 Campochiaro Corrado 4 De Luca Giacomo 4 Gheorghiu Ana Maria 5 Lazzaroni Maria Grazia 6 7 Khanna Dinesh 8 on behalf of the EUSTAR Young Investigator Group 1 Royal National Hospital for Rheumatic Diseases, Bath, UK 2 Department of Pharmacy & Pharmacology, University of Bath, Bath, UK 3 Systemic Immune-Mediated Diseases Unit, Department of Medicine IV, Fernando Fonseca Hospital, Amadora, Portugal 4 Unit of Immunology, Rheumatology, Allergy and Rare Diseases (UnIRAR), IRCCS San Raffaele Hospital, Vita-Salute San Raffaele University, Milan, Italy 5 Internal Medicine and Rheumatology, Cantacuzino Hospital, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania 6 Rheumatology and Clinical Immunology Unit, ASST Spedali Civili of Brescia, Brescia, Italy 7 Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy 8 Scleroderma Program, Division of Rheumatology, University of Michigan, Ann Arbor, MI, USA John D Pauling, Royal National Hospital for Rheumatic Diseases, Upper Borough Walls, Bath BA1 1RL, UK. Email: [email protected] 11 2019 2397198319886496 19 3 2019 4 10 2019 © The Author(s) 2019 2019 SAGE Publications Patient-reported outcome instruments provide valuable insight into disease-related morbidity known only to the patient and complement more objective outcome tools in the clinical trial setting. They are of particular importance in systemic sclerosis owing to the challenges around defining disease activity, the episodic nature of many disease-specific manifestations and the paucity of validated objective surrogate outcome measures for use in clinical trials. Early clinical trials of systemic sclerosis often incorporated legacy patient-reported outcome instruments, but the last 20 years has witnessed the emergence of several scleroderma-specific instruments that are now being routinely used alongside other outcomes in systemic sclerosis clinical trials. More recently, the value of patient-reported outcomes has been highlighted by their prominence in the American College of Rheumatology Combined Response Index for Systemic Sclerosis that has been utilized as the primary endpoint of recent clinical trials of early diffuse systemic sclerosis. This review considers the role and performance of the various patient-reported outcome instruments utilized in systemic sclerosis clinical trials, the current positioning of patient-reported outcome instruments within clinical trial endpoint models across the range of systemic sclerosis disease manifestations and, where applicable, we shall highlight areas for future research. Patient-reported outcome instruments clinical trials systemic sclerosis Raynaud’s phenomenon outcome measures National Institute of Arthritis and Musculoskeletal and Skin Diseases https://doi.org/10.13039/100000069 K24 AR06312 edited-state corrected-proof Declaration of conflicting interests J.P. has consultancy relationships with Actelion and Boehringer Ingelheim. None of the other authors declared any relevant conflicts of interest. Funding The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This work was funded in part by the NIH/NIAMS K24 AR06312 grant held by D.K. ORCID iD John D Pauling https://orcid.org/0000-0002-2793-2364
Keywords
- clinical trials
- outcome measures
- Patient-reported outcome instruments
- Raynaud’s phenomenon
- systemic sclerosis
ASJC Scopus subject areas
- Immunology and Allergy
- Rheumatology
- Immunology