Initial Results from a Pilot Comparative Effectiveness Study of 3 Methotrexate-based Consensus Treatment Plans for Juvenile Localized Scleroderma.
CLINICAL TRIALS
DISEASE ACTIVITY SCORE
METHOTREXATE
PEDIATRIC RHEUMATIC DISEASES
SCLERODERMA
Journal
The Journal of rheumatology
ISSN: 0315-162X
Titre abrégé: J Rheumatol
Pays: Canada
ID NLM: 7501984
Informations de publication
Date de publication:
01 08 2020
01 08 2020
Historique:
accepted:
30
09
2019
pubmed:
17
10
2019
medline:
1
9
2021
entrez:
17
10
2019
Statut:
ppublish
Résumé
To perform a comparative effectiveness feasibility study in juvenile localized scleroderma (LS), using standardized treatment regimens (consensus treatment plans; CTP). 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). 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. 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.
Identifiants
pubmed: 31615917
pii: jrheum.190311
doi: 10.3899/jrheum.190311
doi:
Substances chimiques
Methotrexate
YL5FZ2Y5U1
Types de publication
Journal Article
Multicenter Study
Observational Study
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1242-1252Subventions
Organisme : NIAMS NIH HHS
ID : RC2 AR058934
Pays : United States