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Development and validation of a preliminary definition of minimal disease activity in patients with juvenile
Silvia Magni-Manzoni1, Nicolino Ruperto, Angela Pistorio
1Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico S. Matteo, Pavia, Italy.
Objective:
To develop and validate a definition of minimal disease activity (MDA) in patients with juvenile idiopathic arthritis (JIA).
Methods:
The clinical charts of JIA patients followed over a 16-year period were reviewed to identify visits with high disease activity and MDA, defined on the basis of therapeutic decisions made by the attending physician. For each JIA activity measure recorded at the time of the visit, the cutoff value that best identified states of MDA was calculated by means of the area under the receiver operating characteristic curve analysis. A definition of MDA for oligoarthritis and polyarthritis was set up after testing the relative power of each variable in a multivariate analysis. Validation procedures included assessment of discriminant and construct validity.
Results:
The definition that resulted from the analyses led to establish that a state of MDA could be defined as the presence of a physician global assessment < or =2.5 cm and a swollen joint count of 0 in patients with oligoarthritis; and as the presence of a physician global assessment < or =3.4 cm, a parent global assessment < or =2.1 cm, and a swollen joint count < or =1 in patients with polyarthritis. Validation procedures demonstrated that the MDA definition had good discriminant and construct validity in the context of both observational studies and controlled trials.
Conclusion:
We developed a preliminary definition of MDA in patients with JIA that represents a useful treatment target state and is proposed for inclusion as an outcome measure in future observational studies and clinical trials in patients with JIA.
Insights
Researchers defined minimal disease activity (MDA) for juvenile idiopathic arthritis (JIA) patients. This new MDA definition provides a valuable treatment target for JIA clinical trials and studies.
Area of Science:
- Pediatric Rheumatology
- Clinical Trial Design
- Disease Activity Measurement
Background:
- Juvenile idiopathic arthritis (JIA) management requires objective measures of disease activity.
- Defining minimal disease activity (MDA) is crucial for setting treatment targets in JIA.
Purpose of the Study:
- To develop and validate a precise definition for minimal disease activity (MDA) in patients diagnosed with juvenile idiopathic arthritis (JIA).
Main Methods:
- Retrospective chart review of JIA patients over 16 years to identify high disease activity and MDA states.
- Utilized receiver operating characteristic (ROC) curve analysis to determine optimal cutoff values for JIA activity measures.
- Employed multivariate analysis to establish MDA definitions for oligoarticular and polyarticular JIA, followed by discriminant and construct validity assessments.
Main Results:
- Established MDA criteria for oligoarthritis: physician global assessment ≤2.5 cm and 0 swollen joints.
- Defined MDA for polyarthritis: physician global assessment ≤3.4 cm, parent global assessment ≤2.1 cm, and ≤1 swollen joint.
- Validated MDA definition demonstrated strong discriminant and construct validity in observational studies and controlled trials.
Conclusions:
- A preliminary definition of MDA in JIA has been successfully developed.
- This MDA definition serves as a practical treatment target for JIA patients.
- Proposed inclusion of this MDA definition as a key outcome measure in future JIA research and clinical trials.
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