@article{42d7de96bc144b088388ec1e2bfdb0c6,
title = "Initial Results from a Pilot Comparative Effectiveness Study of 3 Methotrexate-based Consensus Treatment Plans for Juvenile Localized Scleroderma",
abstract = "Objective. To perform a comparative effectiveness feasibility study in juvenile localized scleroderma (LS), using standardized treatment regimens (consensus treatment plans; CTP). Methods. A prospective, multicenter 1-year pilot observational cohort study was performed by Childhood Arthritis and Rheumatology Research Alliance (CARRA) LS workgroup members. Patients with active, moderate to severe juvenile LS were treated with one of 3 CTP: methotrexate alone, or in combination with intravenous (30 mg/kg/dose for 3 mos) or oral corticosteroids (2 mg/kg/ day tapered by 48 weeks). Results. Fifty patients, with demographics typical for juvenile LS, were enrolled, and 44 (88%) completed the study. Most had extracutaneous involvement. Patients improved in all 3 CTP, 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 3 CTP. 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. Response differences were associated with baseline disease activity level, LS subtype, skin disease extent, and extracutaneous involvement. Conclusion. This study demonstrates the feasibility of conducting juvenile LS comparative effectiveness studies. The CTP were found to be safe, effective, and tolerable. Our assessments performed well. Because 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.",
keywords = "Clinical trials, Disease activity score, Methotrexate, Pediatric rheumatic diseases, Scleroderma",
author = "Li, {Suzanne C.} and Torok, {Kathryn S.} and Rabinovich, {C. Egla} and Fatma Dedeoglu and Becker, {Mara L.} and Ferguson, {Polly J.} and Hong, {Sandy D.} and Ibarra, {Maria F.} and Katie Stewart and Elena Pope and Higgins, {Gloria C.} and Laxer, {Ronald M.} and Thomas Mason and Fuhlbrigge, {Robert C.} and Tracy Andrews",
note = "Funding Information: From the Joseph M. Sanzari Children{\textquoteright}s Hospital, Hackensack University Medical Center, Hackensack Meridian School of Medicine at Seton Hall University, Nutley, New Jersey; UPMC Children{\textquoteright}s Hospital of Pittsburgh, Pittsburgh, Pennsylvania; Duke University School of Medicine, Durham, North Carolina; Boston Children{\textquoteright}s Hospital, Boston, Massachusetts; Children{\textquoteright}s Mercy Hospital, Kansas City, Missouri; University of Iowa Carver College of Medicine; University of Iowa Stead Family Children{\textquoteright}s Hospital, Iowa City, Iowa; Children{\textquoteright}s Medical Center of Dallas and UT Southwestern, Dallas, Texas; The Ohio State University, Columbus, Ohio; Mayo Clinic, Rochester, Minnesota; University of Colorado–Denver and Children{\textquoteright}s Hospital Colorado, Denver, Colorado; Rutgers University, School of Public Health, Newark, New Jersey, USA; University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) Legacy Registry was supported by a grant from the US National Institute of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health (NIH) under award number RC2AR058934. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. CARRA, Friends of CARRA, the Arthritis Foundation, and the Duke Clinical Research Institute also supported the CARRA Legacy Registry. This study was funded primarily by an innovative research grant from the Arthritis foundation (PI: SL). CARRA provided additional funding for data analysis (publication grant, PI: SL) and in-kind resources to support management and maintenance of the registry for the study, online meetings, and face-to-face meetings at annual meetings of CARRA and the American College of Rheumatology. Support for biobanking was provided by independent funding from The Nancy Taylor Foundation for Chronic Diseases Inc. (PI: KST, Children{\textquoteright}s Hospital of Pittsburgh Pediatric Scleroderma Fund). S.C. Li, MD, PhD, Joseph M. Sanzari Children{\textquoteright}s Hospital, Hackensack University Medical Center, Hackensack Meridian School of Medicine at Seton Hall University; K.S. Torok, MD, UPMC Children{\textquoteright}s Hospital of Pittsburgh; C.E. Rabinovich, MD, MPH, Duke University School of Medicine; F. Dedeoglu, MD, Boston Children{\textquoteright}s Hospital; M.L. Becker, MD, MSCE, Children{\textquoteright}s Mercy Hospital; P.J. Ferguson, MD, University of Iowa Carver College of Medicine; S.D. Hong, MD, University of Iowa Stead Family Children{\textquoteright}s Hospital; M.F. Ibarra, MD, Children{\textquoteright}s Mercy Hospital; K. Stewart, MD, Children{\textquoteright}s Medical Center of Dallas and UT Southwestern; E. Pope, MD, University of Toronto and The Hospital for Sick Children; G.C. Higgins, MD, PhD, The Ohio State University; R.M. Laxer, MD, University of Toronto and The Hospital for Sick Children; T. Mason II, MD, Mayo Clinic; R.C. Fuhlbrigge, MD, PhD, University of Colorado–Denver, and Children{\textquoteright}s Hospital Colorado; T. Andrews, MA, Rutgers University, School of Public Health. Address correspondence to Dr. S.C. Li, Hackensack University Medical Center, Imus PC337, 30 Prospect Ave., Hackensack, New Jersey 07061, USA. E-mail: suzanne.li@hackensackmeridian.org Accepted for publication September 30, 2019. Publisher Copyright: {\textcopyright} 2020 Journal of Rheumatology. All rights reserved.",
year = "2020",
month = aug,
day = "1",
doi = "10.3899/jrheum.190311",
language = "English (US)",
volume = "47",
pages = "1242--1252",
journal = "Journal of Rheumatology",
issn = "0315-162X",
publisher = "Journal of Rheumatology",
number = "8",
}