Background Juvenile localized scleroderma (JLS) is a chronic inflammatory fibrosing disorder with numerous disease subtypes, results in deep tissue atrophy, limited functional capacity. Treatment of jLS is still contraversal, ranges from topical skin management to immunosuppressive therapy (IST).
Objectives To estimate the resons for IST and its efficacy in JLS,
Methods We detected antibodies to collagen I-IV types (AbC) (IFA, IMTEC); antinuclear antibodies (IFI, Orgentesc) anti dsDNA, anti-topoisomerase (SCL-70), anticentromere (ACA), antinuclear antibodies (ANA), rheumatoid factor (RF); serum level of gyaluronic acid (GA) (IFA, Corgenix), fibronectin (FN) (IFI, Technoclone) in 150 JLS, 35 juvenile systemic sclerosis (JSS) patients and 100 healthy control children.Efficacy of IST was estimated retrospectively in 385 JLS pts. by skin score, skin thickness (Durometr), joint immobility, laboratory tests.
Results Penicillanine (PA) monotherapy 8-10 mg/rg/d for 3-4 years we used in 76 pts (40 - single plague morphea, 15 - generalized scleroderma (scld), 10 - “en coup de sabre” JLS, 11 - linear morphea. PA was effective in 98% of - single plague morphea, 20% of generalized and linear scld, 2% of “en coup de sabre” (ECDS) pts.
PA 8-10 mg/kg/d for 3-5 years + Prednisone (Pr) 1mg/kg/day for 6-10 weeks, then taped and stopped in 12 mo. Were given to 135 JLS pts (46 - linear scld, 45 - hemitype scld, 44 - generalized scld, 30 - ECDS. PA+Pr was effective in 90% of linear, generalized and- hemitype scld pts, 48% ECDS pts.
Methotrexate (MTX) monotherapy 10-15 mg/m2/wk for 2-5 years we used in 100 JLS pts (58 - linear scld, 25 - generalized scld, 17 - ECDS. MTX was benefit in 92% linear and generalized scld pts, 50% ECDS.
MTX 10-15 mg/m2/wk for 2-5 years + Pr 1mg/kg/day for 8-10 weeks, then taped and stopped in 12 mo. We used in 75 JLS (35 hemitype scld pts, linear scld – 25 pts, 1 with ECDS). MTX +Pr was effective in 96% of hemitype & linear scld pts, in 60% ECDS.
Average efficacy of IST for different JLS subtypes varied from 2 to 60% in ECDS scld, from 90% to 96% in hemitype & linear scld, decreasing in pts with disease duration more than 34 months. JLS pts received IST have stopped skin progression and musle atrophy, skin fibrosis and joint contractures have been partly reversed. In contrast to JLS pts (N=120) without IST, observed at our department in 1960-1980, who showed progression of fibrotic skin and joint damage in 70% and organ involvement in 5% of cases in 3 year follow up.
Conclusions Our study shows that autoantibodies and some markers of fibrosis are increased in JLS pts, that makes indroduction of IST proven in scld children without organ involvement, but rapid spread progression of skin,musle and joint desease. Indications for IST regimes for each scld subtype are not clear now. Our experience suggests that pts with single plaque morphea have good response to PA monotherapy, the possibility to avoide cytostatics is important. The most resistant is ECDS type.
Zulian F, Martini G, Vallongo C, et al. Methotrexate in juvenile localized scleroderma: a randomised, double-blind, placebo-controlled trial. Arthritis Rheum. 2011;63(7),1988-2006.
Disclosure of Interest None declared