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

  • Silvia Rosina
  • Giulia Camilla Varnier
  • Marta Mazzoni
  • Stefano Lanni
  • Clara Malattia
  • Angelo Ravelli
Inflammatory Muscle Disease (I Lundberg, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Inflammatory Muscle Disease

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.

Keywords

Juvenile dermatomyositis Idiopathic inflammatory myopathies Outcome measures Randomized clinical trials Disease remission 

Notes

Compliance with Ethical Standards

Conflict of Interest

The authors declare that they have no conflict of interest.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

References

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  • Silvia Rosina
    • 1
  • Giulia Camilla Varnier
    • 1
  • Marta Mazzoni
    • 1
  • Stefano Lanni
    • 2
  • Clara Malattia
    • 1
    • 2
  • Angelo Ravelli
    • 1
    • 2
  1. 1.Università degli Studi di GenovaGenoaItaly
  2. 2.Clinica Pediatrica e ReumatologiaIstituto Giannina GasliniGenoaItaly

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