Childhood Arthritis and Rheumatology Research Alliance Biologic DMARD Consensus Treatment Plans for Refractory Moderately Severe Juvenile Dermatomyositis.

OBJECTIVE: The objective was to develop consensus treatment plans (CTPs) for patients with refractory moderately severe juvenile dermatomyositis (JDM) treated with biologic disease-modifying antirheumatic drugs (bDMARDs). METHODS: The Biologics Workgroup of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) JDM Research Committee used case-based surveys, consensus framework, and nominal group technique to produce bDMARD CTPs for patients with refractory moderately severe JDM. RESULTS: Four bDMARD CTPs were proposed: TNF-alpha inhibitor (adalimumab or infliximab), abatacept, rituximab, and tocilizumab. Each CTP has different options for dosing and/or route. Among 76 respondents, consensus was achieved for the proposed CTPs (93% [67/72]) as well as for patient characteristics, assessments, outcome measures, and follow up. By weighted average, respondents indicated that they would most likely use rituximab followed by abatacept, TNF-alpha inhibitor, and tocilizumab. CONCLUSION: CTPs for the use of bDMARDs in refractory moderately severe JDM were developed using consensus methodology. The implementation of the bDMARD CTPs will lay the groundwork for registry-based prospective comparative effectiveness studies.

Association of p155/140 Autoantibody With Loss of Nailfold Capillaries but not Generalized Lipodystrophy: A Study of Ninety-Six Children With Juvenile Dermatomyositis.

OBJECTIVE: Myositis-specific antibodies (MSAs) facilitate grouping children with juvenile dermatomyositis (DM) into distinct phenotypes. The first aim of this study was to investigate the link between anti-p155/140 and lipodystrophy as determined by dual x-ray absorptiometry (DXA) assessment of fat distribution. The second aim was to examine the relationship between anti-p155/140 and damage to the nailfold capillary system. METHODS: Children with juvenile DM followed for a minimum of 5 years were included. The study population was divided into 3 groups (anti-p155/140, other MSA, and MSA negative). Lipodystrophy was assessed by physician assessment and DXA fat distribution (trunk-to-leg fat ratio). Documentation of nailfold capillary end row loops (ERLs) was obtained at diagnosis. RESULTS: A total of 96 subjects (44% anti-p155/140, 23% other MSA, 33% MSA negative) were included. There was no significant difference in age, disease activity scores, or lipodystrophy between the 3 groups. The trunk-to-leg fat ratios were similar among the 3 groups at different time points. However, the anti-p155/140 group had significantly decreased ERL counts (P = 0.006) at baseline as well as a prolonged duration of untreated disease at diagnosis (P = 0.027). Also, the anti-p155/140 group had fewer patients with a monophasic disease course than the other 2 groups (P = 0.008). CONCLUSION: Generalized lipodystrophy frequency was equivalent in all 3 groups based on physician assessments and trunk-to-leg fat ratios. The anti-p155/140 group had a greater loss of ERLs, suggesting that this MSA may impact the vascular component of juvenile DM.

International Consensus for the Dosing of Corticosteroids in Childhood-Onset Systemic Lupus Erythematosus With Proliferative Lupus Nephritis.

OBJECTIVE: To develop a standardized steroid dosing regimen (SSR) for physicians treating childhood-onset systemic lupus erythematosus (SLE) complicated by lupus nephritis (LN), using consensus formation methodology. METHODS: Parameters influencing corticosteroid (CS) dosing were identified (step 1). Data from children with proliferative LN were used to generate patient profiles (step 2). Physicians rated changes in renal and extrarenal childhood-onset SLE activity between 2 consecutive visits and proposed CS dosing (step 3). The SSR was developed using patient profile ratings (step 4), with refinements achieved in a physician focus group (step 5). A second type of patient profile describing the course of childhood-onset SLE for ≥4 months since kidney biopsy was rated to validate the SSR-recommended oral and intravenous (IV) CS dosages (step 6). Patient profile adjudication was based on majority ratings for both renal and extrarenal disease courses, and consensus level was set at 80%. RESULTS: Degree of proteinuria, estimated glomerular filtration rate, changes in renal and extrarenal disease activity, and time since kidney biopsy influenced CS dosing (steps 1 and 2). Considering these parameters in 5,056 patient profile ratings from 103 raters, and renal and extrarenal course definitions, CS dosing rules of the SSR were developed (steps 3-5). Validation of the SSR for up to 6 months post-kidney biopsy was achieved with 1,838 patient profile ratings from 60 raters who achieved consensus for oral and IV CS dosage in accordance with the SSR (step 6). CONCLUSION: The SSR represents an international consensus on CS dosing for use in patients with childhood-onset SLE and proliferative LN. The SSR is anticipated to be used for clinical care and to standardize CS dosage during clinical trials.