Biologic therapies for refractory juvenile dermatomyositis: five years of experience of the Childhood Arthritis and Rheumatology Research Alliance in North America

Springer Science and Business Media LLC - Tập 15 - Trang 1-8 - 2017
CH Spencer1, K Rouster-Stevens2, H Gewanter3, G Syverson4, R Modica5, K Schmidt6, H Emery7, C Wallace7, S Grevich7, K Nanda7, YD Zhao7, S Shenoi7, S Tarvin8, S Hong9, C Lindsley10, JE Weiss11, M Passo12, K Ede13, A Brown14, K Ardalan15, W Bernal16, ML Stoll17, B Lang18, R Carrasco19, C Agaiar20, L Feller21, H Bukulmez22, R Vehe23, H Kim24, H Schmeling25, D Gerstbacher26, M Hoeltzel27, B Eberhard28, R Sundel29, S Kim16, AM Huber18, A Patwardhan30
1Nationwide Children’s Hospital and Ohio State University, Columbus, USA
2Emory University School of Medicine, Atlanta, USA
3Pediatric and Adolescent Health Partners, Richmond, USA
4University of Wisconsin-Madison, Madison, USA
5University of Florida, Gainesville, USA
6University of Louisville, Louisville, USA
7Seattle Children’s Hospital, University of Washington, Seattle, USA
8Riley Hospital for Children, Indiana University Medical Center, Indianapolis, India
9University of Iowa Health Care, Iowa City, USA
10University of Kansas Medical Center, Kansas City, USA.
11Sanzari Children’s Hospital, Hackensack University Medical Center, Hackensack, USA
12University of South Carolina, Charleston, USA
13Phoenix Children's Hospital, Phoenix, USA
14Texas Children’s Hospital, Houston, USA
15Lurie Children’s Hospital, Chicago, USA
16University of California San Francisco, San Francisco, USA
17University of Alabama at Birmingham, Birmingham, USA
18WK Health Center and Dalhousie University, Halifax, Canada
19Dell Children’s Hospital, Austin, USA
20Children’s Hospital of The Kings Daughter, Norfolk, USA
21Inland Rheumatology, Waterville, USA
22Metro Health Medical Center and Case Western Reserve University, Cleveland, USA
23University of Minnesota, Minneapolis, USA
24National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, USA
25Alberta Children’s Hospital, Cumming School of Medicine, University of Calgary, Calgary, Canada
26Lucille Packard Children’s Hospital, Stanford University, Stanford, USA
27Mott Children’s Hospital, University of Michigan, Ann Arbor, USA
28Cohen Children’s Medical Center of New York, New york, USA
29Boston Children’s Hospital and Harvard Medical School, Boston, USA
30School of Medicine, University of Missouri, Columbia, USA

Tóm tắt

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). 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. 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. 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

Tài liệu tham khảo

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