TY - JOUR
T1 - Innovative Research Design to Meet the Challenges of Clinical Trials for Juvenile Dermatomyositis
AU - Rosina, Silvia
AU - Varnier, Giulia Camilla
AU - Mazzoni, Marta
AU - Lanni, Stefano
AU - Malattia, Clara
AU - Ravelli, Angelo
PY - 2018/5/1
Y1 - 2018/5/1
N2 - 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.
AB - 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.
KW - Disease remission
KW - Idiopathic inflammatory myopathies
KW - Juvenile dermatomyositis
KW - Outcome measures
KW - Randomized clinical trials
UR - http://www.scopus.com/inward/record.url?scp=85045261870&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85045261870&partnerID=8YFLogxK
U2 - 10.1007/s11926-018-0734-4
DO - 10.1007/s11926-018-0734-4
M3 - Review article
AN - SCOPUS:85045261870
VL - 20
JO - Current Rheumatology Reports
JF - Current Rheumatology Reports
SN - 1523-3774
IS - 5
M1 - 29
ER -