Related Experiment Video
Updated: Jul 1, 2026
02:28
Measuring Psoriasis Severity at Home
Published on: March 1, 2024
Outcome measures in rheumatologic clinical trials and systemic sclerosis
1Geffen School of Medicine, University of California, Los Angeles, CA, USA. defurst@mednet.ucla.edu
Rheumatology (Oxford, England)
|September 17, 2008
Summary
Outcome measures for systemic sclerosis (SSc) clinical trials are being refined. Validated measures include skin scores and pulmonary function tests, while others like echocardiograms for pulmonary hypertension require further research.
Area of Science:
- Rheumatology
- Clinical Trial Methodology
- Systemic Sclerosis (SSc) Research
Background:
- The Outcome Measures in Rheumatologic Clinical Trials (OMERACT) organization aims to enhance measurement tools for rheumatic diseases.
- Systemic sclerosis (SSc) presents complex challenges in assessing treatment response due to multi-organ involvement.
Purpose of the Study:
- To review the current status of outcome measurement instruments for SSc in clinical trials.
- To identify validated, under-validation, and not-yet-validated measures for SSc and its complications.
Main Methods:
- Literature review and consensus-building among rheumatologists, epidemiologists, and statisticians.
- Evaluation of existing and emerging measurement tools for various SSc manifestations.
Main Results:
- Validated measures for SSc include the modified Rodnan skin score, Raynaud's condition score, forced vital capacity, and specific markers for scleroderma renal crisis.
- The gastrointestinal questionnaire is nearing validation, while measures for pulmonary arterial hypertension (e.g., echocardiogram, CT scan, PFTs, 6-min walk test, MRI) are still under investigation.
Conclusions:
- Several reliable outcome measures exist for SSc, but further validation is needed for specific complications like pulmonary arterial hypertension.
- Continued development and validation of measurement tools are crucial for advancing SSc clinical trial efficacy and patient care.
Related Concept Videos
01:22Rheumatic Heart Disease II: Clinical Manifestations and Diagnostic Studies
The key clinical manifestations of Rheumatic heart disease (RHD) include several distinct cardiac symptoms.Carditis, a hallmark of acute rheumatic fever, involves inflammation of the heart's endocardium, myocardium, and pericardium. Chronic RHD often results from recurrent episodes of carditis. Its symptoms include the following:Murmurs are caused by valvular damage, especially to the mitral and aortic valves. Mitral stenosis or regurgitation is common, with characteristic heart murmurs...
01:11Clinical Trials: Overview
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...