Background: The prognosis of children with juvenile dermatomyositis (JDM) has improved remarkably since the\n1960ââ?¬â?¢s with the use of corticosteroid and immunosuppressive therapy. Yet there remain a minority of children\nwho have refractory disease. Since 2003 the sporadic use of biologics (genetically-engineered proteins that\nusually are derived from human genes) for inflammatory myositis has been reported. In 2011ââ?¬â??2016 we investigated our\ncollective experience of biologics in JDM through the Childhood Arthritis and Rheumatology Research Alliance (CARRA).\nMethods: The JDM biologic study group developed a survey on the CARRA member experience using biologics\nfor Juvenile DM utilizing Delphi consensus methods in 2011ââ?¬â??2012. The survey was completed online by the\nCARRA members interested in JDM in 2012. A second survey was similarly developed that provided more\nopportunity to describe their experiences with biologics in JDM in detail and was completed by CARRA members\nin Feb 2013. During three CARRA meetings in 2013ââ?¬â??2015, nominal group techniques were used for achieving\nconsensus on the current choices of biologic drugs. A final survey was performed at the 2016 CARRA meeting.\nResults: One hundred and five of a potential 231 pediatric rheumatologists (42%) responded to the first survey in\n2012. Thirty-five of 90 had never used a biologic for Juvenile DM at that time. Fifty-five of 91 (denominators vary)\nhad used biologics for JDM in their practice with 32%, 5%, and 4% using rituximab, etanercept, and infliximab,\nrespectively, and 17% having used more than one of the three drugs. Ten percent used a biologic as monotherapy, 19%\na biologic in combination with methotrexate (mtx), 52% a biologic in combination with mtx and corticosteroids, 42% a\ncombination of a biologic, mtx, corticosteroids (steroids), and an immunosuppressive drug, and 43% a combination of a\nbiologic, IVIG and mtx. The results of the second survey supported these findings in considerably more detail with\nmultiple combinations of drugs used with biologics and supported the use of rituximab, abatacept, anti-TNFÃ?± drugs,\nand tocilizumab in that order. One hundred percent recommended that CARRA continue studying biologics for JDM.\nThe CARRA meeting survey in 2016 again supported the study and use of these four biologic drug groups.\nConclusions: Our CARRA JDM biologic work group developed and performed three surveys demonstrating that\npediatric rheumatologists in North America have been using multiple biologics for refractory JDM in numerous scenarios\nfrom 2011 to 2016. These survey results and our consensus meetings determined our choice of four biologic therapies\n(rituximab, abatacept, tocilizumab and anti-TNFÃ?± drugs) to consider for refractory JDM treatment when indicated and to\nevaluate for comparative effectiveness and safety in the future.
Loading....