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Current status of clinical outcome measures in inclusion body myositis: a systematised review

Roy, Bhaskar; Lucchini, Matteo; Lilleker, James B; Goyal, Namita A; Naddaf, Elie; Adler, Brittany; Alfano, Lindsay N; ... International Myositis Assessment and Clinical Studies (IMACS); + view all (2023) Current status of clinical outcome measures in inclusion body myositis: a systematised review. Clinical and Experimental Rheumatology , 41 (2) pp. 370-378. 10.55563/clinexprheumatol/ifacv3. Green open access

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Abstract

OBJECTIVES: Sporadic inclusion body myositis (IBM) is a debilitating idiopathic inflammatory myopathy (IIM) which affects hand function, ambulation, and swallowing. There is no approved pharmacological therapy for IBM, and there is a lack of suitable outcome measure to assess the effect of an intervention. The IBM scientific interest group under IMACS reviewed the previously used outcome measures in IBM clinical studies to lay the path for developing a core set of outcome measures in IBM. METHODS: In this systematised review, we have extracted all outcome measures reported in IBM clinical studies to determine what measures were being used and to assess the need for optimising outcome measures in IBM. RESULTS: We found 13 observational studies, 17 open-label clinical trials, and 15 randomised control trials (RCTs) in IBM. Six-minute walk distance, IBM-functional rating scale (IBM-FRS), quantitative muscle testing, manual muscle testing, maximal voluntary isometric contraction testing, and thigh muscle volume measured by MRI were used as primary outcome measures. Twelve different outcome measures of motor function were used in IBM clinical trials. IBM-FRS was the most used measure of functionality. Swallowing function was reported as a secondary outcome measure in only 3 RCTs. CONCLUSIONS: There are inconsistencies in using outcome measures in clinical studies in IBM. The core set measures developed by the IMACS group for other IIMs are not directly applicable to IBM. As a result, there is an unmet need for an IBM-specific core set of measures to facilitate the evaluation of new potential therapeutics for IBM.

Type: Article
Title: Current status of clinical outcome measures in inclusion body myositis: a systematised review
Location: Italy
Open access status: An open access version is available from UCL Discovery
DOI: 10.55563/clinexprheumatol/ifacv3
Publisher version: https://doi.org/10.55563/clinexprheumatol/ifacv3
Language: English
Additional information: This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Humans, Myositis, Inclusion Body, Myositis, Muscle, Skeletal, Walking, Outcome Assessment, Health Care
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases
URI: https://discovery.ucl.ac.uk/id/eprint/10166627
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