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Toward a Treat-to-Target Strategy in Juvenile Dermatomyositis: What Are the Suitable Targets and Optimal Timing of
Jayne M MacMahon1,2, Angelo Ravelli3,4, Ana Isabel Rebollo-Giménez5,6
1Division of Rheumatology, Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.
Insights
This study analyzed treatment response times in juvenile dermatomyositis (JDM) patients. Findings support developing treat-to-target (T2T) strategies by providing timelines for achieving remission in JDM care.
Area of Science:
- Pediatric Rheumatology
- Autoimmune Diseases
- Clinical Trial Design
Background:
- Juvenile dermatomyositis (JDM) is a rare autoimmune condition affecting children.
- The treat-to-target (T2T) strategy is gaining traction in pediatric rheumatology for managing chronic conditions.
- An international expert initiative is developing T2T recommendations specifically for JDM.
Purpose of the Study:
- To determine the time to treatment response in juvenile dermatomyositis (JDM) patients.
- To provide data supporting the development of a treat-to-target (T2T) strategy for JDM.
- To inform clinical practice regarding expected timelines for JDM treatment outcomes.
Main Methods:
- Retrospective review of physician-confirmed JDM cases at two tertiary care centers.
- Analysis of demographic and clinical data within the first two years post-diagnosis.
- Utilized Kaplan-Meier survival curves to define time to specific treatment outcomes.
Main Results:
- 187 JDM patients were included, with a mean age of 8 years at diagnosis.
- Muscle enzyme normalization averaged 3 months; muscle remission averaged 6 months post-treatment.
- Skin remission was observed within 12 months; time to inactive disease varied by center (8.8-10.3 months).
Conclusions:
- This study offers real-world data on treatment response timelines in JDM.
- The findings can inform the establishment of achievable targets for a T2T strategy in JDM.
- Provides evidence-based timelines to guide therapeutic adjustments in JDM management.
Objective:
Juvenile dermatomyositis (JDM) is a rare autoimmune condition. The treat-to-target strategy has garnered interest in pediatric rheumatology. It is based on defining clear therapeutic targets, with frequent disease activity monitoring, and adjustment of the treatments if targets are not met within a defined time frame. Recently, an international task force of experts launched an initiative aimed at the development of recommendations for the adoption of treat-to-target strategy in JDM. This study was done to support those recommendations. We aimed to determine the time-to-treatment response in patients with JDM, to better inform the development of a treat-to-target strategy in JDM.
Methods:
This is a retrospective review of patients with a physician-confirmed diagnosis of JDM, observed at two tertiary care centers-the Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini (Gaslini), and The Hospital for Sick Children (SickKids). Demographic and clinical data were obtained on all patients with JDM during the first two years following diagnosis. Kaplan-Meier survival curves were used to determine time to outcome definitions.
Results:
A total of 187 patients were identified across two sites; the mean age of diagnosis was 8 years. On average, patients with JDM achieved normalization of muscle enzymes and muscle remission three months and six months after treatment initiation, respectively. Skin remission occurred within 12 months after starting treatment. Time to reach inactive disease varied between the sites, with median time being 10.3 months (Gaslini) and 8.8 months (SickKids).
Conclusion:
This study provides real-world data for potential timelines to target with a treat-to-target strategy for JDM.
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