Background/Purpose:   Juvenile localized scleroderma (jLS) is a chronic inflammatory and fibrosing disease that causes severe morbidity, including growth defects, cosmetic deformities, seizures and arthropathy. Optimal therapy is not known. The LS Children’s Arthritis and Rheumatology Research Alliance (CARRA) subgroup has been working towards conducting comparative effectiveness studies with the aim of improving long-term outcome. We report our initial findings from a pilot study to evaluate the feasibility of conducting such studies.

Methods:    

We conducted a multi-center 12 month prospective observational cohort study of jLS subjects treated with one of 3 methotrexate (MTX)-based standardized treatment regimens (consensus treatment plans, CTPs), chosen by the treating physician. Each CTP included MTX administered for 1 year; two included corticosteroids (CS), either intravenous (IV) for 3 months or oral for 48 weeks (tapering schedule).  The CTP options are based upon best available evidence and reflect the current treatment practices of CARRA members  (Li et al., Arthritis Rheum 2012; 64:1175).  Data were entered into a web-based registry (InForm), including activity and damage assessments, global assessments (GA), adverse events (AE), and medications. 

Results:

The target enrollment (50 subjects) was reached. Median age at enrollment was 13 years, majority of subjects were Caucasian (92%), female (70%), had the linear subtype (82%) and had never been treated with MTX or CS (82%). Fourty percent of subjects had a head lesion, 72% had extracutaneous involvement.

            Participating sites showed strong CTP preferences, with 50% choosing only 1 and 40% 2 CTPs. Half the subjects were enrolled into MTX + IV CS, 14 into MTX alone, and 11 into MTX + oral CS CTPs. Ninety percent of subjects were followed for at least 6 months, 80% completed the full 12 months.

            Among subjects followed for at least 6 months, 49% were considered to have a major and 31% a moderate improvement in activity level compared to baseline. Subjects in the MTX + CS CTPs were more likely than those in the MTX alone CTP to be rated as having a major improvement (57% vs 22%). Thirty two percent of subjects had a major deviation from their starting CTP with the most common reasons being AE (28%) and lack of response (28%). The AEs most commonly associated with deviations were severe gastrointestinal symptoms, mood issues, and infections. There was 1 SAE: one subject required hospitalization for viral gastroenteritis. Mycophenolate mofetil and/or IV CS were the most commonly employed therapies when there was a lack of response.   Subjects that deviated from initial CTP had a higher physician GA of disease activity and patient/parent GA of disease impact at baseline compared to those that did not. 

Conclusion:   Our pilot study shows the feasibility of conducting jLS comparative effectiveness studies across multiple centers. Despite a high rate of deviation from the CTPs, the majority of subjects were rated as having moderate or major improvement. Centers showed strong biases in their treatment preferences highlighting the need to have sufficient site variability and CTPs to enable comparative effectiveness studies.

Funded by Arthritis Foundation, DCRI, NIAMS, and Friends of CARRA

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/initial-results-of-a-pilot-juvenile-localized-scleroderma-jls-comparative-effectiveness-study/