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.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Pediatr+Rheumatol+Online+J
2017 ; 15
(1
): 50
Nephropedia Template TP
gab.com Text
Twit Text FOAVip
Twit Text #
English Wikipedia
Biologic therapies for refractory juvenile dermatomyositis: five years of
experience of the Childhood Arthritis and Rheumatology Research Alliance in North
America
#MMPMID28610606
Spencer CH
; Rouster-Stevens K
; Gewanter H
; Syverson G
; Modica R
; Schmidt K
; Emery H
; Wallace C
; Grevich S
; Nanda K
; Zhao YD
; Shenoi S
; Tarvin S
; Hong S
; Lindsley C
; Weiss JE
; Passo M
; Ede K
; Brown A
; Ardalan K
; Bernal W
; Stoll ML
; Lang B
; Carrasco R
; Agaiar C
; Feller L
; Bukulmez H
; Vehe R
; Kim H
; Schmeling H
; Gerstbacher D
; Hoeltzel M
; Eberhard B
; Sundel R
; Kim S
; Huber AM
; Patwardhan A
Pediatr Rheumatol Online J
2017[Jun]; 15
(1
): 50
PMID28610606
show ga
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-TNF? 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-TNF? 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.