Treatment of Juvenile Linear Scleroderma
All patients under 18 with active, potentially disfiguring or disabling juvenile linear scleroderma should be treated with methotrexate 15 mg/m²/week (oral or subcutaneous) combined with systemic corticosteroids during the initial 2-3 months as bridge therapy. 1, 2
Referral and Initial Assessment
Refer all suspected cases to a specialized pediatric rheumatology center given the disease rarity (3.4 cases per million children annually). 1, 3
Perform comprehensive skin examination documenting linear sclerotic plaques, active inflammatory lesions (violaceous borders, erythema), and architectural changes using photography or diagrams. 3
Use the Localized Scleroderma Cutaneous Assessment Tool (LoSCAT) at baseline and follow-up, which includes the LoSSI for activity scoring and LoSDI for damage assessment. 2, 3
Screen for extracutaneous involvement including arthritis, myositis, uveitis, neurologic manifestations, and growth disturbances, as these can present before skin disease. 4
First-Line Systemic Treatment Algorithm
For active linear scleroderma (the most common subtype in children): 4
Start methotrexate 15 mg/m²/week as a single oral or subcutaneous dose. 1, 2
Simultaneously initiate systemic corticosteroids using one of two accepted regimens: 1
- Oral prednisone 1-2 mg/kg/day for 2-3 months with gradual tapering, OR
- Pulsed intravenous methylprednisolone 30 mg/kg with various schedules
Continue corticosteroids for the first 3 months as adjunctive "bridge therapy" while methotrexate takes effect. 1
Maintain methotrexate for at least 12 months after achieving acceptable clinical improvement before considering tapering, as prolonged remission off medication is more likely with extended treatment duration. 1, 2, 3
Treatment Duration and Monitoring
Monitor using LoSCAT scores at each visit to quantify response to therapy. 2, 3
Check for methotrexate side effects including nausea, headache, and transient hepatotoxicity at regular intervals. 2, 3
Consider withdrawing methotrexate only once the patient achieves remission and has been off corticosteroids for at least 1 year. 1
Recognize that recurrence rates are 25-48% in the first years after treatment discontinuation, necessitating long-term follow-up. 3, 4
Second-Line Treatment for Refractory or Intolerant Patients
Use mycophenolate mofetil 500-1000 mg/m² for severe disease or in patients who are methotrexate-refractory or methotrexate-intolerant. 1, 2, 3
Consider biologics (TNF or IL-6 inhibitors), tacrolimus, or cyclophosphamide for resistant cases or CNS involvement, though high-level evidence is lacking. 1
Treatment for Isolated Circumscribed Lesions
For superficial, isolated plaques without deep tissue involvement (cosmetic concern only): 2, 3
Medium-dose UVA1 phototherapy improves skin softness and reduces thickness, though requires prolonged maintenance with cumulative radiation exposure concerns. 1, 2
Topical imiquimod decreases skin thickening by upregulating interferons that inhibit collagen production. 1, 2, 3
Critical Pitfalls to Avoid
Do not delay systemic treatment. Patients must be treated in the "therapeutic window" before significant irreversible fibrosis develops, as mean disease duration at diagnosis is often 8 months. 5, 6
Do not underestimate the importance of aggressive treatment. Skin disease is associated with high physical and psychological morbidity, permanent functional disability, and growth disturbances in children. 1, 2, 4
Do not discontinue treatment prematurely. Inadequate treatment duration leads to relapse; maintain therapy for at least 12 months after clinical improvement. 2, 3
Do not rely solely on skin examination for activity assessment. Extracutaneous manifestations (arthritis, myositis, uveitis, neurologic involvement) require separate monitoring and indicate disease activity. 3, 4
Adjunctive Management
- Physical therapy, plastic surgery, and orthopedic management are essential to address residual limitations and improve quality of life after achieving disease control. 7