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J Rheumatol. 2019 Oct 15. pii: jrheum.190311. doi: 10.3899/jrheum.190311. [Epub ahead of print]

Initial Results from a Pilot Comparative Effectiveness Study of Three Methotrexate-Based Consensus Treatment Plans for Juvenile Localized Scleroderma.

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From Hackensack University Medical Center, Hackensack, Meridian School of Medicine at Seton Hall University, NJ; UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA; Duke University School of Medicine, Durham, NC; Boston Children's Hospital Boston, MA; Children's Mercy, Kansas City, MO; University of Iowa Carver College of Medicine, Iowa City, IA; University of Iowa Stead Family Children's Hospital, Iowa City, IA; Children's Medical Center of Dallas and UT Southwestern, Dallas, TX; University of Toronto and The Hospital for Sick Children, Toronto, Canada; The Ohio State University, Columbus, OH; Mayo Clinic, Rochester, MN; University of Colorado- Denver and Children's Hospital Colorado, Denver, CO; Rutgers University, School of Public Health, Newark, NJ. Financial support received from Arthritis Foundation Innovative Research Grant and Childhood Arthritis and Rheumatology Research Alliance. SCL has received consulting fees from Bristol Myers Squibb (<$10,000) and royalties from UpToDate. RML is a consultant for Alexion, Sanofi, Eli Lilly Canada, Novartis, and Sobi. Address correspondence to Suzanne C. Li, MD PhD Hackensack University Medical Center; Imus PC337, 30 Prospect Ave, Hackensack, NJ 07061.



To perform a comparative effectiveness feasibility study in juvenile localized scleroderma (jLS), using standardized treatment regimens (consensus treatment plans, CTPs).


A prospective, multi-center 1-year pilot observational cohort study was performed by Childhood Arthritis and Rheumatology Research Alliance (CARRA) localized scleroderma workgroup members. Active, moderate to severe jLS patients were treated with one of three CTPs: methotrexate alone, or in combination with intravenous (30 mg/kg/dose for 3 months) or oral corticosteroids (2 mg/kg/day tapered off by 48 weeks).


Fifty patients, with demographics typical for jLS, were enrolled, and 44 (88%) completed the study. Most had extracutaneous involvement. Patients improved in all three CTPs, with >75% having a major or moderate level of improvement compared to baseline. Damage accrued in some patients. Major deviations from prescribed regimen resulted from medication intolerance (n = 6, 14%) or treatment failure (n = 11, 25%); failures occurred in all three CTPs. Significant responses to treatment were demonstrated by LS skin scoring measures and overall physician assessments, with differences in response level identified in some patient subsets. Baseline disease activity level, LS subtype, skin disease extent, and extracutaneous involvement were associated with response differences.


This study demonstrates the feasibility of conducting jLS comparative effectiveness studies. The CTPs were found to be safe, effective, and tolerable. Our assessments performed well. As damage is common and may progress despite effective control of activity, we recommend initial treatment efficacy be evaluated primarily by activity measures. Potential confounders for response were identified that warrant further study.


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