[2009] [SAT0541] LONG TERM OUTCOME OF 14 PATIENTS WITH SEVERE FORM OF JUVENILE LOCALIZED SCLERODERMA

T. Avcin¹, T. Vesel¹, N. Toplak¹, V. Dragos^{2 1}Department of Allergology, Rheumatology and Clinical Immunology; ²Department of Dermatology, University Childrens Hospital Ljubljana, University Medical Center, Ljubljana, Slovenia

Background: Juvenile localized scleroderma (JLS) is a rare disorder in children whose manifestations are mostly confined to the skin and subdermal tissues. No controlled trials have demonstrated effectiveness of any modality of treatment in patients with JLS and treatment remains a challenge. There is increasing evidence that some new treatment protocols are beneficial in JLS.
Objectives: To determine impact of treatment on the long term outcome of JLS.
Methods: A retrospective cohort study of all children with severe form of JLS followed at the University Children's Hospital Ljubljana from January 2003 to December 2008. Inclusion criteria were (i) onset of JLS prior to the patient's 18th birthday, (ii) evidence of active disease at the time of diagnosis and (iii) severe form of the disease (multilocular, rapidly progressive, contractures and/or associated arthritis). All patients were classified according to the preliminary classification criteria of JLS [1]. Patients were assessed at the time of diagnosis, at the beginning of new therapy, at remission and at the last follow-up visit. Standardized data collection form was used to record the clinical characteristics of the patients including evaluation of skin lesions (progression/regression or development of new skin lesions), development of extra-cutaneous manifestations, long term damage at the last clinic visit (functional and cosmetic effects) and laboratory data.
Results: Fourteen patients with severe JLS were identified during the study period. Nine were girls and 5 boys with mean age at disease onset 6.6±3.4 years (range: 2.4-12.9 years). Six (43%) patients had linear scleroderma, 4 (29%) generalized morphea, 2 (14%) circumscribed morphea and 2 (14%) mixed morphea. All patients had active and progressive skin lesions. Extracutaneous manifestations were present in two patients including arthritis (n=1) and headache (n=1). Laboratory investigations showed positive antinuclear antibodies in 9 (64%), positive antiphospholipid antibodies in 2 (14%) and positive B. Burgdorferi immunoserology in 2 (14%) patients. Mean age at the start of treatment was 7.8±3.6 years. Seven patients received methotrexate p.o. ± pulse methylprednisolone i.v. (3 patients were previously unresponsive to antibiotics), 7 patients received penicillin or ceftriaxon i.v. ± short-term systemic steroids for one month and 3 patients received topical therapy. Remission was achieved in 6/7 (86%) patients treated with methotrexate p.o. ± pulse methylprednisolone i.v., 3/7 (43%) patients treated with antibiotics i.v. ± short-term systemic steroids and 1/3 (33%) patient with topical therapy. Time to remission in patients treated with methotrexate was significantly shorter than in patients treated with i.v. antibiotics (4.3 months vs. 2.5 years). Three patients stopped methotrexate after a mean time of 16.8 months and had no relapse.
Conclusion: There are no accurate data on the natural history and optimal management of JLS. Methotrexate p.o. appears to be effective therapy in our cohort of patients with severe form of JLS and we propose a larger, multicenter study investigating impact of treatment on the long term outcome of JLS.
References:

1. Laxer RM and Zulian F. Localized scleroderma. Curr Opin Rheumatol 2006;18:606-13.