Session Type: Abstract Submissions (ACR)
Purpose: To evaluate clinical assessment tools and standardized treatment regimens (consensus treatment plans, CTPs) developed for juvenile localized scleroderma (jLS)
jLS is a chronic, inflammatory and fibrosing disease often associated with severe morbidity in the growing child. Over 20% of the jLS subjects enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry have joint contractures and >10% have a limb length difference. A major issue with evaluating the relative efficacy of the many reported treatments has been the lack of agreed upon assessments and criteria for evaluating treatment response. Our CARRA subgroup recently developed jLS disease assessments, response criteria, and CTPs that reflect current treatment practices of CARRA members, and the performance characteristics of these tools and feasibility of conducting comparative effectiveness treatment studies will be evaluated in a pilot study.
We are conducting a prospective, multi-center (10 CARRA sites), 1 year observational study of a cohort of 50 jLS patients beginning treatment with one of three methotrexate (MTX)-based CTPs (MTX alone, MTX with oral corticosteroids [CS], or MTX with intravenous CS), with study data entered into the existing web-based CARRA Registry. The treating physician determines which CTP to use. Collected data includes scoring of clinical activity and damage parameters, scoring of joint/limb morbidity; medication log; adverse events; physician global assessments (PGA) for activity, damage, and response to treatment; and patient and parent quality of life measures. There is an optional patient sample collection substudy. A reliability workshop meeting was held at study onset to review and evaluate the reliability of scoring the clinical assessments with 14 study physicians scoring 13 jLS patient volunteers two times.
Results: There was moderate inter-rater reliability for clinical activity and damage scoring (Kendall’s coefficient 0.628 and 0.508, respectively), and high intra-rater reliability (average Spearman’s rho 0.783 for activity, 0.743 for damage). Some parameters appeared more problematic, with lower reliability scores found for the activity parameters of lesion warmth, violaceous color, and skin thickness of lesion edge, and for the damage parameters hypopigmentation, and dermal atrophy. Since study enrollment began in 12/2012, 18 subjects from 9 sites have been enrolled. Nearly all subjects have agreed to participate in the optional biosample collection
We have currently achieved 36% of our target enrollment of 50 jLS subjects into our pilot study. This study enables evaluation and refinement of clinical tools for assessing disease state and treatment response based upon patient data. This study will allow us to evaluate feasibility issues related to the developed CTPs and with conducting comparative effectiveness treatment studies. Because the biorepository being developed is linked to extensive clinical data in a prospective registry, these samples potentially will allow the identification of biomarkers associated with good versus poor outcome, as well as better understanding of disease pathogenesis.
S. C. Li,
K. S. Torok,
M. L. Becker,
R. C. Fuhlbrigge,
G. C. Higgins,
S. D. Hong,
M. F. Ibarra,
R. M. Laxer,
T. G. Mason II,
M. G. Punaro,
C. E. Rabinovich,
jannsen; Abbott, UCB,
K. G. Stewart,
K. M. Wittkowski,
« Back to 2013 ACR/ARHP Annual Meeting
ACR Meeting Abstracts - https://acrabstracts.org/abstract/a-pilot-study-of-juvenile-localized-scleroderma-jls-consensus-treatment-plans/