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Outcome Measures in Pediatric Rheumatic Disease
Sarah Ringold1, Alessandro Consolaro2, Stacy P Ardoin3
1Seattle Children's, 4800 Sand Point Way Northeast, Seattle, WA 98115, USA.
Rheumatic Diseases Clinics of North America
|October 12, 2021
Summary
Developing reliable outcome measures is key for pediatric rheumatic disease trials. This review details clinician-reported measures for juvenile idiopathic arthritis, juvenile dermatomyositis, childhood-onset systemic lupus erythematosus, and pediatric vasculitis.
Area of Science:
- Pediatric Rheumatology
- Clinical Trial Methodology
- Outcome Measurement
Background:
- Reliable outcome measures are essential for advancing pediatric rheumatic disease research and clinical care.
- Current research requires validated tools to accurately track disease progression and treatment efficacy in children.
- Existing measures need to be responsive to changes in disease state, activity, and damage.
Purpose of the Study:
- To review and outline clinician-reported, disease-specific outcome measures for key pediatric rheumatic diseases.
- To highlight the development of validated composite outcome measures through consensus-based and direct measurement approaches.
- To provide a resource for researchers and clinicians involved in pediatric rheumatic disease trials.
Main Methods:
- Literature review of validated, composite outcome measures.
- Focus on clinician-reported and disease-specific instruments.
- Inclusion of measures for juvenile idiopathic arthritis, juvenile dermatomyositis, childhood-onset systemic lupus erythematosus, and pediatric vasculitis.
Main Results:
- Several validated, composite outcome measures have been developed for specific pediatric rheumatic diseases.
- These measures utilize a combination of consensus-based and direct measurement approaches.
- The review identifies clinician-reported tools crucial for trial conduct.
Conclusions:
- Validated, clinician-reported outcome measures are critical for effective pediatric rheumatic disease research.
- The development of these measures facilitates observational and interventional trials.
- These tools have the potential to directly transform care for children with rheumatic diseases.
Keywords:
Childhood-onset lupusJuvenile dermatomyositisJuvenile idiopathic arthritisOutcome measurePediatricsVasculitisMore Related Videos
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