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Table 1 Working jLS Study Entry Criteria: Inclusion, Exclusion, and Active Disease criteria

From: Developing comparative effectiveness studies for a rare, understudied pediatric disease: lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study

Inclusion Criteria:
 1. Localized scleroderma diagnosed by a Pediatric Rheumatologist or Pediatric Dermatologist according to Padua Preliminary Classification criteria; these criteria exclude eosinophilic fasciitis [23]
 2. Fulfill active disease criteria:
  a. Either at least one item from Active disease criteria Group 1, or two from Group 2
 3. Moderate to high disease severity that warrants systemic therapy in the opinion of the treating physician
  a. Includes all subtypes that involve deeper tissue, extensive skin involvement, and/or extracutaneous involvement
 4. Age < 18 years at onset of disease
 5. Age < 21 years at onset of treatment
Exclusion criteria:
 1. Treated with systemic corticosteroids in the prior 2 weeks
 2. Treated with methotrexate or mycophenolate mofetil within the prior 3 monthsa
 3. Another defined systemic rheumatic disease (e.g., systemic sclerosis)
 4. Intolerance to study medications
Working Active Disease criteria (used for Pilot Consensus Treatment Plan study)a
Group 1:
 1. New lesion(s) within the prior 3 months, documented by cliniciana
 2. Extension of an existing lesion within the prior 3 months, documented by cliniciana
  a. Lesion extension observed in serial photographs or tracings, or detecting > 30% difference in lesion size (maximum length x width).
 3. Documentation of active or progressive deep tissue involvementa
  a. Can be by clinical examination, photographs, MRI, or ultrasound
 4. Erythema of moderate or severe level in lesion or an erythematous lesion border
  a. Erythema scoring level based upon LS Scoring Atlas
 5. Violaceous lesion or border color
  a. Can range from lilac ring to deep violaceous color
Group 2:
 1. Patient or parent report of new lesion OR extension of existing lesion occurring within the prior 3 monthsa
  a. This criterion ONLY applies for new patients (i.e., first visit to clinician’s office).
 2. Erythema of mild level
  a. Erythema scoring level based upon LS Scoring Atlas
 3. Moderate or severe induration of lesion bordera
  a. Assessed according to modified Rodnan Skin Scoring (mRSS) levels [15]
 4. Tactile warmth of the lesion
  a. Examiner appreciation of temperature difference based upon comparison to control site (unaffected contralateral site if available).
 5. Worsening hair loss in scalp, eyebrow, or eyelashes; documented by cliniciana
 6. Elevated creatine kinase level in the absence of other sourcea
 7. Lesion biopsy showing active disease (based upon pathologist report. Typically would be presence of lymphocytes, plasma cells, eosinophils, or other white blood cell)
  1. Study entry criteria used for the jLS Consensus Treatment Plan (CTP) Pilot study
  2. These criteria were previously developed [20] for the purpose of directing comparative effectiveness studies in jLS and were not intended to qualify or disqualify patients for any specific treatment. The LS Scoring Atlas was generated by the LS workgroup of CARRA and contains photographs of patient lesions demonstrating the different visible scored features; > 80% consensus agreement by workgroup members was required for the photograph to be included in the atlas [20]
  3. adenotes criteria that were modified or deleted as a result of this study—see Table 2