Background/Purpose   Juvenile localized scleroderma (jLS) often causes severe morbidity in the developing child, including growth defects and disfigurement. Optimal therapy is not known.  The LS Children’s Arthritis and Rheumatology Research Alliance (CARRA) subgroup has been working towards improving long-term outcome for these patients. Towards this end, we have developed standardized treatment regimens based upon best available evidence and consensus methodology (consensus treatment plans, CTPs), and clinical tools to use in comparative effectiveness treatment studies. We are currently conducting a pilot study to evaluate the feasibility of conducting jLS comparative effectiveness treatment studies. Additional analyses will include evaluating performance characteristics of developed tools.

Methods Fifteen physicians from 10 CARRA centers have been conducting a prospective observational cohort study of jLS subjects initiating systemic immunosuppressive treatment. Inclusion criteria include diagnosis of jLS by pediatric rheumatologist or dermatologist, and presence of active disease according to delineated activity criteria generated by the group. Exclusion criteria include treatment with methotrexate (MTX) within prior 3 months or corticosteroids (CS) within prior 2 weeks. Subjects were treated with one of three MTX-based CTPs (MTX alone, MTX with intravenous CS (IV CS), or MTX with oral CS), determined by treating physician, and evaluated at 6 visits over 1 year.  At the start of the study, a workshop meeting was held to standardize evaluation. 

Results

The target enrollment (50 subjects) was reached. All sites enrolled subjects, with enrollment taking approximately 23 months to complete following study initiation at the first site. Subjects were enrolled in all 3 CTPs, with half enrolled in MTX + IV CS CTP. Over 40% of subjects deviated from their initial treatment regimen, with persistent activity a frequent reason. Over 80% of subjects agreed to participate in the optional sample collection and banking sub study.

Study Subject Features
Gender	35 Females (73%)
Age at study entry, median (range)	12.7 yr (3 – 21 yr)
Race	43 White 1 Black 2 Asian 2 unknown
Ethnicity	39 non-Hispanic: 9 Hispanics
Treatment Regimen MTX alone MTX+intravenous CS MTX + oral CS	12 (25.5%) 24 (51%) 11 (23%)
Number who deviated from starting CTP among subjects who completed at least 2 study visits	15/34 (44%)
Agreed to participate in sample collection	38/46 (82%)
Completed 1 year of study visits	13 (26%)
Dropped out from study	2 (4%)

Conclusion

This is the first study to explore the feasibility of conducting comparative effectiveness treatment studies in jLS.  We achieved our target enrollment of 50 subjects, with subjects enrolled in all 3 standardized treatment regimens. Biological samples have been collected from the majority of subjects, which will enable future translational studies.  This study will enable us to evaluate and refine clinical tools needed for treatment studies based upon study data, and identify issues related to conducting jLS treatment studies. Further analyses of these data once completed will also include clinical effectiveness and tolerability of the 3 different treatment regimens in LS subjects.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/a-pilot-study-to-evaluate-the-feasibility-of-conducting-juvenile-localized-scleroderma-comparative-effectiveness-treatment-studies/