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Innovative Research Design to Meet the Challenges of Clinical Trials for Juvenile Dermatomyositis

  • Inflammatory Muscle Disease (I Lundberg, Section Editor)
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Abstract

Purpose of Review

This paper aims to provide a summary of the recent therapeutic advances and the latest research on outcome measures for clinical trials in juvenile dermatomyositis (JDM).

Recent Findings

Recent randomized controlled trials (RCTs) have demonstrated the superiority of the combination of prednisone with methotrexate over other conventional therapies and the potential effectiveness of rituximab in refractory cases. A multinational project has led to develop new criteria for the definition of minimal, moderate, and major improvement in future JDM clinical trials. This effort has been paralleled by the establishment of criteria for clinically inactive disease. The validation of the first composite disease activity score for JDM is in progress.

Summary

The new outcome measures will increase the reliability of assessment of clinical response in JDM clinical trials and foster future multinational RCTs aimed to investigate novel treatment strategies for refractory forms of JDM.

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Authors and Affiliations

  1. Università degli Studi di Genova, Genoa, Italy

    Silvia Rosina, Giulia Camilla Varnier, Marta Mazzoni, Clara Malattia & Angelo Ravelli

  2. Clinica Pediatrica e Reumatologia, Istituto Giannina Gaslini, Via G. Gaslini 5, 16147, Genoa, Italy

    Stefano Lanni, Clara Malattia & Angelo Ravelli

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  1. Silvia Rosina
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  2. Giulia Camilla Varnier
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  3. Marta Mazzoni
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  4. Stefano Lanni
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  5. Clara Malattia
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  6. Angelo Ravelli
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Correspondence to Angelo Ravelli.

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This article is part of the Topical Collection on Inflammatory Muscle Disease

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Rosina, S., Varnier, G.C., Mazzoni, M. et al. Innovative Research Design to Meet the Challenges of Clinical Trials for Juvenile Dermatomyositis. Curr Rheumatol Rep 20, 29 (2018). https://doi.org/10.1007/s11926-018-0734-4

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