Biologic therapies for refractory juvenile dermatomyositis: five years of experience of the Childhood Arthritis and Rheumatology Research Alliance in North America
نویسندگان
چکیده
BACKGROUND The prognosis of children with juvenile dermatomyositis (JDM) has improved remarkably since the 1960's with the use of corticosteroid and immunosuppressive therapy. Yet there remain a minority of children who have refractory disease. Since 2003 the sporadic use of biologics (genetically-engineered proteins that usually are derived from human genes) for inflammatory myositis has been reported. In 2011-2016 we investigated our collective experience of biologics in JDM through the Childhood Arthritis and Rheumatology Research Alliance (CARRA). METHODS The JDM biologic study group developed a survey on the CARRA member experience using biologics for Juvenile DM utilizing Delphi consensus methods in 2011-2012. The survey was completed online by the CARRA members interested in JDM in 2012. A second survey was similarly developed that provided more opportunity to describe their experiences with biologics in JDM in detail and was completed by CARRA members in Feb 2013. During three CARRA meetings in 2013-2015, nominal group techniques were used for achieving consensus on the current choices of biologic drugs. A final survey was performed at the 2016 CARRA meeting. RESULTS One hundred and five of a potential 231 pediatric rheumatologists (42%) responded to the first survey in 2012. Thirty-five of 90 had never used a biologic for Juvenile DM at that time. Fifty-five of 91 (denominators vary) had used biologics for JDM in their practice with 32%, 5%, and 4% using rituximab, etanercept, and infliximab, respectively, and 17% having used more than one of the three drugs. Ten percent used a biologic as monotherapy, 19% a biologic in combination with methotrexate (mtx), 52% a biologic in combination with mtx and corticosteroids, 42% a combination of a biologic, mtx, corticosteroids (steroids), and an immunosuppressive drug, and 43% a combination of a biologic, IVIG and mtx. The results of the second survey supported these findings in considerably more detail with multiple combinations of drugs used with biologics and supported the use of rituximab, abatacept, anti-TNFα drugs, and tocilizumab in that order. One hundred percent recommended that CARRA continue studying biologics for JDM. The CARRA meeting survey in 2016 again supported the study and use of these four biologic drug groups. CONCLUSIONS Our CARRA JDM biologic work group developed and performed three surveys demonstrating that pediatric rheumatologists in North America have been using multiple biologics for refractory JDM in numerous scenarios from 2011 to 2016. These survey results and our consensus meetings determined our choice of four biologic therapies (rituximab, abatacept, tocilizumab and anti-TNFα drugs) to consider for refractory JDM treatment when indicated and to evaluate for comparative effectiveness and safety in the future. Significance and Innovations This is the first report that provides a substantial clinical experience of a large group of pediatric rheumatologists with biologics for refractory JDM over five years. This experience with biologic therapies for refractory JDM may aid pediatric rheumatologists in the current treatment of these children and form a basis for further clinical research into the comparative effectiveness and safety of biologics for refractory JDM.
منابع مشابه
Treatment approaches to juvenile dermatomyositis (JDM) across North America: The Childhood Arthritis and Rheumatology Research Alliance (CARRA) JDM Treatment Survey.
OBJECTIVE There are a number of different approaches to the initial treatment of juvenile dermatomyositis (JDM). We assessed the therapeutic approaches of North American pediatric rheumatologists to inform future studies of therapy in JDM. METHODS A survey describing clinical cases of JDM was sent to pediatric rheumatologists. The cases described children with varying severity of typical dise...
متن کاملClinical characteristics of children with juvenile dermatomyositis recruited within the first 7 months of the CARRAnet registry
Purpose Performing quality clinical and translational research in juvenile dermatomyositis (JDM) is difficult due to the rarity of the disease. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) initiated a multicenter observational cohort study to create a foundational clinical database for the major rheumatic diseases of childhood, including JDM. Initial data from the JDM coho...
متن کاملChildhood Arthritis and Rheumatology Research Alliance consensus clinical treatment plans for juvenile dermatomyositis with skin predominant disease
BACKGROUND Juvenile dermatomyositis (JDM) is the most common form of the idiopathic inflammatory myopathies in children. A subset of children have the rash of JDM without significant weakness, and the optimal treatments for these children are unknown. The goal of this study was to describe the development of consensus clinical treatment plans (CTPs) for children with JDM who have active skin ra...
متن کاملDiagnostic evaluation and medication usage in a cohort of subjects with juvenile dermatomyositis from the CARRAnet registry
Purpose Juvenile dermatomyositis (JDM) is a rare disease which has been difficult to evaluate objectively due to the low incidence of disease. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) initiated a multicenter observational registry to create a clinical database for the major rheumatic diseases of childhood, including JDM. Initial data from the JDM cohort (prevalent and ...
متن کاملPres-FINAL-2009: Pediatric rheumatology practitioners experience with biologics in juvenile dermatomyositis: survey results
Introduction Biologic therapy is increasingly prescribed in rheumatologic disorders. Juvenile dermatomyositis (JDM), the most common inflammatory myopathy in children, can be challenging to manage in a subset of patients. There are multiple reports of cytokine involvement in JDM. There is a paucity of information regarding the use of biologics for JDM among pediatric rheumatology practitioners,...
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عنوان ژورنال:
دوره 15 شماره
صفحات -
تاریخ انتشار 2017