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Treating juvenile dermatomyositis to target: Paediatric Rheumatology European Society/Childhood Arthritis and
Angelo Ravelli1, Silvia Rosina2, Jayne M MacMahon3
1Direzione Scientifica, IRCCS Istituto Giannina Gaslini, Genoa, Italy; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.
Insights
New guidelines offer a treat-to-target strategy for juvenile dermatomyositis (JDM) to improve patient outcomes. The recommendations focus on achieving inactive disease within 12 months for better JDM management.
Area of Science:
- Pediatric Rheumatology
- Autoimmune Diseases
- Clinical Practice Guidelines
Background:
- Juvenile dermatomyositis (JDM) presents suboptimal response to current therapies in a significant patient proportion.
- Recent advancements have improved JDM prognosis, yet treatment optimization remains crucial.
Purpose of the Study:
- To develop evidence-based, consensus-driven recommendations for a treat-to-target strategy in JDM.
- To establish clear treatment goals and timelines for managing JDM.
Main Methods:
- Systematic literature review and retrospective chart review informed provisional recommendations.
- An international Task Force of 34 experts, including patients and parents, discussed and voted on recommendations.
- Consensus was achieved on 7 principles and 12 recommendations with ≥80% agreement.
Main Results:
- Inactive disease is the preferred target for JDM, achievable within 12 months.
- Interim targets include clinical improvement at 6 weeks and 3 months, and normalized muscle strength at 6 months.
- High-dose glucocorticoids are foundational initially, with a plan for tapering within 12 months via immunomodulatory therapy.
Conclusions:
- Recommendations for a treat-to-target approach in JDM were developed by a multidisciplinary Task Force.
- While evidence requires expansion through future research, these guidelines aim to optimize JDM outcomes.
- Implementation in clinical practice is expected to enhance treatment effectiveness for JDM patients.
Objectives:
Despite the recent prognostic improvement, a sizeable proportion of patients with juvenile dermatomyositis (JDM) respond suboptimally to contemporary therapies. This study aimed to develop recommendations for treating JDM to target.
Methods:
A Steering Committee formulated a set of provisional recommendations based on evidence derived from a systematic literature review and a retrospective chart review of patients. These were discussed, amended, and voted on by an international Task Force, including 28 paediatric rheumatologists, 2 specialists in neuromuscular diseases, 1 dermatologist, 1 physical therapist, 1 research nurse, 2 patients with JDM, and 1 parent of a patient with JDM. Items that achieved at least an 80% majority vote were accepted as final recommendations.
Results:
Although the literature review did not reveal trials that compared a treat-to-target strategy with a nonsteered approach, it provided indirect evidence about specific end points that could serve as targets that facilitated development of recommendations. The group reached consensus on 7 overarching principles and 12 recommendations. It was agreed that both patients/parents and treaters should share decisions in setting treatment targets and therapeutic strategies, with inactive disease as the preferred target and minimal disease activity an alternative one. Inactive disease is targeted to be achieved within 12 months after treatment start. Interim targets include minimal and moderate clinical improvement within 6 weeks and 3 months, respectively, and normalisation of muscle strength within 6 months. High-dose glucocorticoids remain fundamental in the initial management, but progressive tapering and discontinuation within 12 months through optimisation of concomitant immunomodulatory therapy was advised. A research agenda was formulated.
Conclusions:
The Task Force developed recommendations for treating JDM to target, being aware that the evidence is not strong and needs to be expanded by future research. Implementation of the recommendations in clinical practice will help to reach optimal outcomes for JDM.
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