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Approaches for estimating minimal clinically important differences in systemic lupus erythematosus
Open accessAbstract
A minimal clinically important difference (MCID) is an important concept used to determine whether a medical intervention improves perceived outcomes in patients. Prior to the introduction of the concept in 1989, studies focused primarily on statistical significance. As most recent clinical trials in systemic lupus erythematosus (SLE) have failed to show significant effects, determining a clinically relevant threshold for outcome scores (that is, the MCID) of existing instruments may be critical for conducting and interpreting meaningful clinical trials as well as for facilitating the establishment of treatment recommendations for patients. To that effect, methods to determine the MCID can be divided into two well-defined categories: distribution-based and anchor-based approaches. Distribution-based approaches are based on statistical characteristics of the obtained samples. There are various methods within the distribution-based approach, including the standard error of measurement, the standard deviation, the effect size, the minimal detectable change, the reliable change index, and the standardized response mean. Anchor-based approaches compare the change in a patient-reported outcome to a second, external measure of change (that is, one that is more clearly understood, such as a global assessment), which serves as the anchor. Finally, the Delphi technique can be applied as an adjunct to defining a clinically important difference. Despite an abundance of methods reported in the literature, little work in MCID estimation has been done in the context of SLE. As the MCID can help determine the effect of a given therapy on a patient and add meaning to statistical inferences made in clinical research, we believe there ought to be renewed focus on this area. Here, we provide an update on the use of MCIDs in clinical research, review some of the work done in this area in SLE, and propose an agenda for future research.
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Metadata
- Authors
- Rai, S. K., Yazdany, J., Fortin, P. R., & Aviña-Zubieta, J. A.
- Affiliations
- University of British Columbia (CA)
- University of California, San Francisco (US)
- Université Laval (CA)
- Corresponding author
- Aviña-Zubieta, J Antonio
- Venue
- Arthritis Research & Therapy
- Published
- 2015-06-03
- Volume
- 17
- Issue
- 1
- Pages
- 143-143
- ISSN
- 1478-6354 (ISSN-L)
- PMC
- PMC4453215
- OpenAlex
- W1487863225
- Cited by
- 251
- References
- 56
- Citations by year
- 2026: 12 · 2025: 15 · 2024: 35 · 2023: 34 · 2022: 29 · 2021: 38 · 2020: 30 · 2019: 20 · 2018: 15 · 2017: 12 · 2016: 9 · 2015: 2
- FWCI
- 1.96
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Arthritis Research & Therapy (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- eScholarship (California Digital Library) (submittedVersion, cc-by)
- Funders
- Université Laval
- National Institutes of Health
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (K23AR060259)
- Topics
- Health Sciences › Medicine › Rheumatology › Systemic Lupus Erythematosus Research
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Social Sciences › Decision Sciences › Management Science and Operations Research › Psychometric Methodologies and Testing
- Keywords
- minimal clinically important difference, systemic lupus erythematosus, patient-reported outcomes, distribution-based methods, anchor-based methods, reliable change index, standardized response mean, minimal detectable change, standard error of measurement, effect size, Delphi method, outcome measures
- MeSH terms
- Disability Evaluation, Female, Humans, Lupus Erythematosus, Systemic, Male, Severity of Illness Index
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