Development of consensus treatment plans for juvenile localized scleroderma: a roadmap toward comparative effectiveness studies in juvenile localized scleroderma

Abstract

Objective: Juvenile localized scleroderma (LS) is a chronic inflammatory skin disorder associated with substantial morbidity and disability. Although a wide range of therapeutic strategies has been reported in the literature, a lack of agreement on treatment specifics and accepted methods for clinical assessment has made it difficult to compare approaches and identify optimal therapy. Our objective was to develop standardized treatment plans, clinical assessments, and response criteria for active, moderate to high severity juvenile LS.

Methods: A core group of pediatric rheumatologists, dermatologists, and a lay advisor was engaged by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) to develop standardized treatment plans and assessment parameters for juvenile LS using consensus methods/nominal group techniques. Recommendations were validated in 2 face-to-face conferences with a larger group of practitioners with expertise in juvenile LS and with the full membership of CARRA, which encompasses the majority of pediatric rheumatologists in the US and Canada.

Results: Consensus was achieved on standardized treatment plans that reflect the prevailing treatment practices of CARRA members. Standardized clinical assessment methods and provisional treatment response criteria were also developed. Greater than 90% of pediatric rheumatologists responding to a survey (66% of CARRA membership) affirmed the final recommendations and agreed to utilize these consensus plans to treat patients with juvenile LS.

Conclusion: Using consensus methodology, we have developed standardized treatment plans and assessment methods for juvenile LS. The high level of support among pediatric rheumatologists will support future comparative effectiveness studies and enable the development of evidence-based guidelines for the treatment of juvenile LS.

Figure 1. Standardization of corticosteroid and methotrexate treatment regimens for juvenile localized scleroderma

Distribution of initial corticosteroid (CS) and methotrexate (MTX) treatment regimens for jLS based upon surveys of CARRA members conducted before (upper circles) and after (lower circles) development of jLS CTP. The size of each wedge is proportional to the number of respondents specifying this regimen. Current Practice: The CS piechart depicts the 59 initial CS regimens specified for treating a jLS linear scleroderma of limb case vignette (15). Each oral CS dose and intravenous (IV) CS dosing regimen is represented by a different color, with the different shades representing differences in duration of treatment and/or tapering schedule. Different CS types and dosing amounts or schedules are separated by tick marks with the different types/doses indicated above the wedge. For the combination CS treatment (IV + P), only the IV dosing regimen is listed, with different shades representing differences in prednisone dose, prednisone duration and/or IV CS duration. Oral prednisone (P) doses are in milligrams per kilogram of body weight per day. The MTX piechart depicts the 58 MTX regimens specified for the first 12 months of treatment for the same linear scleroderma case vignette (15). Each colored wedge represents a different dose, route and/or duration of treatment for the first 12 months of therapy. Different dosing amounts are separated by tick marks with the dose indicated above the wedge. The values of 0.3 to 1.0 represent milligrams (mg) of MTX per kilogram of patient's body weight. The values of 10 to 20/m2 represent mg of MTX per meter squared of patient's surface area. Consensus Treatment Plans: The CS piechart shows the distribution of CS CTPs that CARRA members estimated they would use to treat future jLS patients. The survey did not ask members to specify which of the two IV CS dosing regimens they would use so this wedge is shaded both purple and green. All of the MTX based CTPs specify the same dosing regimen of MTX administered subcutaneously every week for the first 12 months of treatment. This dosing regimen was unanimously agreed upon by jLS core and LS Workgroup members, and was endorsed by 94% of CARRA responders in 2011 survey. Abbreviations: d= day, IV = intravenous, kg = kilograms, mg = milograms, mo = month, P = prednisone; wk = week.