RT Journal Article
SR Electronic
T1 Pilot Study of the Juvenile Dermatomyositis Consensus Treatment Plans: A CARRA Registry Study
JF The Journal of Rheumatology
JO J Rheumatol
FD The Journal of Rheumatology
SP jrheum.190494
DO 10.3899/jrheum.190494
A1 Kuan Liu
A1 George Tomlinson
A1 Ann M.  Reed
A1 Adam M.  Huber
A1 Olli Saarela
A1 Sharon M.  Bout-Tabaku
A1 Megan Curran
A1 Jeffrey A.  Dvergsten
A1 Barbara A.  Eberhard
A1 Lawrence K.  Jung
A1 Susan Kim
A1 Sarah Ringold
A1 Kelly A.  Rouster-Steven
A1 Melissa Tesher
A1 Dawn M.  Wahezi
A1 Brian M.  Feldman
YR 2020
UL http://www.jrheum.org/content/early/2020/10/13/jrheum.190494.abstract
AB Objective To determine the feasibility of comparing the Childhood Arthritis and Rheumatology Research Alliance (CARRA) consensus treatment plans (CTP) in treating moderate new-onset juvenile dermatomyositis (JDM) using the CARRA registry, and to establish appropriate analytic methods to control for confounding by indication and missing data. Methods A pilot cohort of 39 patients with JDM from the CARRA registry was studied. Patients were assigned by the treating physician, considering patient/family preferences, to 1 of 3 CTP: methotrexate (MTX) and prednisone (MP); intravenous (IV) methylprednisolone, MTX, and prednisone (MMP); or IV methylprednisolone, MTX, prednisone, and IV immunoglobulin (MMPI). The primary outcome was the proportion of patients achieving moderate improvement at 6 months under each CTP. Statistical methods including multiple imputation and inverse probability of treatment weighting were used to handle missing data and confounding by indication. Results Patients received MP (n = 13), MMP (n = 18) and MMPI (n = 8). Patients in all CTP had significant improvement in disease activity. Of the 36 patients who remained in our pilot study at 6 months, 16 (44%) of them successfully achieved moderate improvement at 6 months (6/13, 46% for MP; 7/15, 47% for MMP; 3/8, 38% for MMPI). After correcting for confounding, there were no statistically significant pairwise differences between the CTP (P = 0.328–0.88). Conclusion We gained valuable experience and insight from our pilot study that can be used to guide the design and analysis of comparative effectiveness studies using the CARRA registry CTP approach. Our analytical methods can be adopted for future comparative effectiveness studies and applied to other rare disease observational studies.