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

K Ede; A Brown; K Ardalan; W Bernal; C H Spencer; S Hong; M L Stoll; B Lang; K Rouster-Stevens; H Gewanter; R Carrasco; G Syverson; C Agaiar; R Modica; L Feller; K Schmidt; H Bukulmez; H Emery; R Vehe; H Kim; C Wallace; H Schmeling; S Grevich; K Nanda; D Gerstbacher; Y D Zhao; M Hoeltzel; B Eberhard; S Shenoi; S Tarvin; R Sundel; S Kim; C Lindsley; J E Weiss; A M Huber; M Passo; A Patwardhan

doi:10.1186/s12969-017-0174-0

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

Journal article

Open access

Peer reviewed

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

K Ede, A Brown, K Ardalan, W Bernal, C H Spencer, S Hong, M L Stoll, B Lang, K Rouster-Stevens, H Gewanter, …

Pediatric rheumatology online journal, Vol.15(1), pp.50-50

06/13/2017

DOI: 10.1186/s12969-017-0174-0

PMCID: PMC5470177

PMID: 28610606

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Abstract

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 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.

Disease Resistance

Pediatrics - trends

Drug Therapy, Combination - classification

United States - epidemiology

Glucocorticoids - therapeutic use

Biological Therapy - methods

Humans

Dermatomyositis - epidemiology

Male

Methotrexate - therapeutic use

Infliximab - therapeutic use

Dermatomyositis - therapy

Practice Patterns, Physicians' - statistics & numerical data

Rituximab - therapeutic use

Drug Therapy, Combination - methods

Drug Therapy, Combination - trends

Pediatrics - methods

Female

Surveys and Questionnaires

Child

Medication Therapy Management - trends

Antirheumatic Agents - therapeutic use

Etanercept - therapeutic use

Details

Title: Subtitle: Biologic therapies for refractory juvenile dermatomyositis: five years of experience of the Childhood Arthritis and Rheumatology Research Alliance in North America
Creators: K Ede - Phoenix Children's Hospital, Phoenix, AZ, USA
A Brown - Texas Children's Hospital, Houston, TX, USA
K Ardalan - Lurie Children's Hospital, Chicago, IL, USA
W Bernal - University of California, San Francisco, San Francisco, CA, USA
C H Spencer - Nationwide Children's Hospital and Ohio State University, Columbus, OH, USA. spencercharlesh1@gmail.com
S Hong - University of Iowa Health Care, Iowa City, IA, USA
M L Stoll - University of Alabama at Birmingham, Birmingham, AL, USA
B Lang - WK Health Center and Dalhousie University, Halifax, NS, Canada
K Rouster-Stevens - Emory University School of Medicine, Atlanta, GA, USA
H Gewanter - Pediatric and Adolescent Health Partners, Richmond, VA, USA
R Carrasco - Dell Children's Hospital, Austin, TX, USA
G Syverson - University of Wisconsin-Madison, Madison, WI, USA
C Agaiar - Children's Hospital of The Kings Daughter, Norfolk, VA, USA
R Modica - University of Florida, Gainesville, FL, USA
L Feller - Inland Rheumatology, Waterville, ME, USA
K Schmidt - University of Louisville, Louisville, KY, USA
H Bukulmez - Metro Health Medical Center and Case Western Reserve University, Cleveland, OH, USA
H Emery - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
R Vehe - University of Minnesota, Minneapolis, MN, USA
H Kim - National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA
C Wallace - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
H Schmeling - Alberta Children's Hospital, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada
S Grevich - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
K Nanda - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
D Gerstbacher - Lucille Packard Children's Hospital, Stanford University, Stanford, CA, USA
Y D Zhao - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
M Hoeltzel - Mott Children's Hospital, University of Michigan, Ann Arbor, MI, USA
B Eberhard - Cohen Children's Medical Center of New York, New york, USA
S Shenoi - Seattle Children's Hospital, University of Washington, Seattle, WA, USA
S Tarvin - Riley Hospital for Children, Indiana University Medical Center, Indianapolis, IN, India
R Sundel - Boston Children's Hospital and Harvard Medical School, Boston, MA, USA
S Kim - University of California, San Francisco, San Francisco, CA, USA
C Lindsley - University of Kansas Medical Center, Kansas City, KS, USA
J E Weiss - Sanzari Children's Hospital, Hackensack University Medical Center, Hackensack, NJ, USA
A M Huber - WK Health Center and Dalhousie University, Halifax, NS, Canada
M Passo - University of South Carolina, Charleston, SC, USA
A Patwardhan - School of Medicine, University of Missouri, Columbia, MO, USA
Resource Type: Journal article
Publication Details: Pediatric rheumatology online journal, Vol.15(1), pp.50-50
DOI: 10.1186/s12969-017-0174-0
PMID: 28610606
PMCID: PMC5470177
NLM abbreviation: Pediatr Rheumatol Online J
ISSN: 1546-0096
eISSN: 1546-0096
Publisher: England
Grant note: name: Arthritis Foundation of United States
Language: English
Date published: 06/13/2017
Academic Unit: Stead Family Department of Pediatrics; Rheumatology, Allergy, and Immunology
Record Identifier: 9984070832202771

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