TY - JOUR
T1 - Domains and outcome measures for the assessment of limited cutaneous systemic sclerosis
T2 - a scoping review protocol
AU - Lescoat, Alain
AU - Roofeh, David
AU - Townsend, Whitney
AU - Hughes, Michael
AU - Sandler, Robert D
AU - Zimmermann, François
AU - Pauling, John D
AU - Buch, Maya H
AU - Khanna, Dinesh
N1 - Funding Information:
Funding This project has received funding from SRUK (Scleroderma and Raynaud UK) & WSF (World Scleroderma Foundation) (UH&UHR1) and additional support is provided by the National Institutes of Health/NIAMS grant to Dr DK (K24 AR063120). Dr DR was funded by the NIH/NIAMS T32 grant (AR007080). Dr AL was funded by the French network of the University Hospitals HUGO (Hôpitaux Universitaire du Grand Ouest) (AAP JCM2020) and by the 2020 CORECT grant from the Rennes University Hospital (France).
Funding Information:
Actelion pharmaceuticals, JP has received speaker’s honoraria and research grant support (>$10 000) from Actelion pharmaceuticals. JP has undertaken consultancy work for Actelion pharmaceuticals, Sojournix Pharma and Boehringer Ingelheim, MHB has received meeting support from Boehringer Ingelheim, AL, DR, WT, RS and FZ have no conflict of interest, DK is a consultant to Acceleron, Abbvie, Actelion, Amgen, Bayer, BMS, Boehringer Ingelheim, CSL Behring, Corbus, Galapagos, Genentech/Roche, GSK, Horizon, Mitsubishi Tanabe Pharma, Sanofi-Aventis and United Therapeutics. He has stock options in Eicos Sciences, Inc.
Publisher Copyright:
© 2021 The Author(s). Published by Cambridge University Press.
PY - 2021/3/11
Y1 - 2021/3/11
N2 - INTRODUCTION: Limited cutaneous systemic sclerosis (lcSSc) is the most frequent subset of systemic sclerosis. Despite this, lcSSc is not the major focus of clinical studies. The lack of interventional studies in lcSSc is due, in part, to a paucity of relevant outcome measures to effectively evaluate this subset. A combined response index dedicated to lcSSc would facilitate development of well-designed trials and approval of new drugs. The objective of this scoping review is to perform a broad and comprehensive identification of the outcome measures (core set items) within relevant domains, which have been used so far to assess lcSSc.METHODS AND ANALYSIS: The planned scoping review will be based on the approach proposed by Arksey et al and further developed by Levac et al. Development and reporting will follow the Preferred Reporting Items for Systematic Reviews and Meta-Analyses-Extension for Scoping Reviews checklist and guidelines. The development of the search strategy was guided by the concepts of domains and outcomes based on the Outcome Measures in Rheumatology approach and by the different names and definitions of SSc, with a specific emphasis on their occurrence in clinical trial studies. Two databases will be searched: MEDLINE and Embase. Studies in English, published from the year 1988 onwards, will be included, since 1988 corresponds to the publication of LeRoy's first consensus definition of lcSSc. Data will be extracted and analysed using a standardised charting tool.ETHICS AND DISSEMINATION: No ethical approval is required for this study. The results will be submitted to an international peer-reviewed journal and scientific conferences, informing the discussion on which items should be included in a combined response index dedicated to lcSSc (the CRISTAL project: Combined Response Index for Scleroderma Trial Assessing lcSSc).
AB - INTRODUCTION: Limited cutaneous systemic sclerosis (lcSSc) is the most frequent subset of systemic sclerosis. Despite this, lcSSc is not the major focus of clinical studies. The lack of interventional studies in lcSSc is due, in part, to a paucity of relevant outcome measures to effectively evaluate this subset. A combined response index dedicated to lcSSc would facilitate development of well-designed trials and approval of new drugs. The objective of this scoping review is to perform a broad and comprehensive identification of the outcome measures (core set items) within relevant domains, which have been used so far to assess lcSSc.METHODS AND ANALYSIS: The planned scoping review will be based on the approach proposed by Arksey et al and further developed by Levac et al. Development and reporting will follow the Preferred Reporting Items for Systematic Reviews and Meta-Analyses-Extension for Scoping Reviews checklist and guidelines. The development of the search strategy was guided by the concepts of domains and outcomes based on the Outcome Measures in Rheumatology approach and by the different names and definitions of SSc, with a specific emphasis on their occurrence in clinical trial studies. Two databases will be searched: MEDLINE and Embase. Studies in English, published from the year 1988 onwards, will be included, since 1988 corresponds to the publication of LeRoy's first consensus definition of lcSSc. Data will be extracted and analysed using a standardised charting tool.ETHICS AND DISSEMINATION: No ethical approval is required for this study. The results will be submitted to an international peer-reviewed journal and scientific conferences, informing the discussion on which items should be included in a combined response index dedicated to lcSSc (the CRISTAL project: Combined Response Index for Scleroderma Trial Assessing lcSSc).
KW - immunology
KW - internal medicine
KW - rheumatology
U2 - 10.1136/bmjopen-2020-044765
DO - 10.1136/bmjopen-2020-044765
M3 - Article
C2 - 33707273
SN - 2044-6055
VL - 11
JO - BMJ Open
JF - BMJ Open
IS - 3
M1 - e044765
ER -