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

Research output: Contribution to journalReview article

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.

Original languageEnglish
Article number29
JournalCurrent Rheumatology Reports
Volume20
Issue number5
DOIs
Publication statusPublished - May 1 2018

Keywords

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

ASJC Scopus subject areas

  • Rheumatology

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