Arthritis & Rheumatism, Volume 62,
November 2010 Abstract Supplement

Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Atlanta, Georgia November 6-11, 2010.


Localized Scleroderma Therapy: When Would Be a Good Time To Stop?

Arkachaisri1,  Thaschawee, Torok2,  Kathryn S.

KK Women's and Children's Hosp, Singapore, Singapore
Univ of Pittsburgh Med Ctr, Pittsburgh, PA

Background:

Localized Scleroderma (LS) is the most common form of juvenile scleroderma. Its long-term physical and psychological impact continues to cause significant disability. Different therapy options were reported with inconsistent results. Longterm followup (FU) data after the disease is under control is unknown. Combined high dose SC methotrexate and a weaning dose of oral corticosteroids have been shown to be effective in our pediatric LS cohort.

Purpose:

We report a prospective data on the longterm outcome of a single center treatment protocol in a pediatric LS cohort and propose the duration of therapy for LS.

Methods:

LS patients were recruited from the Scleroderma Clinic at the Children's Hospital of Pittsburgh. Patients with active disease, defined as those with erythematous lesions, and/or new lesions, or expansion of existing lesions; were started on oral prednisone 2 mg/kg/day (max 60mg/day) and SC methotrexate (MTX) at 1 mg/kg/week. Prednisone was weaned to reach 1 mg/kg/day by the end of 6–8 weeks, and then further weaned to 0.25 mg/kg/day to complete 12 months of steroid therapy. MTX was continued for a full 24 months of SC therapy with subsequent oral therapy for 12 more months. The disease outcome parameters used to assess effectiveness of therapy were the modified LS Skin Severity Index (mLoSSI) and the physician global assessment of disease activity (PGA). All patients were followed every 4–8 weeks with appropriate therapy adverse reaction monitoring. Flare was defined by the reoccurrence of erythematous lesions, and/or new lesions, or expansion of existing lesions. Kaplan-Meier analysis was used to analyzed recurrence rate and survival-time data.

Results:

Thirty-nine patients were included (70% female, 95% Caucasians). LS Subtypes are–4 plaque morphea, 14 linear scleroderma (Li), 6 en coup de sabre, 3 subcutaneous morphea (SqM), 5 generalized morphea and 7 mixed LS. Median age at onset was 7.9 years (IQR 4.5–11.6). Median duration of FU was 36.9 mo(range 8.7–105.8). Clinical improvement (mLoSSI and PGA) was demonstrated at the median of 1.1 mo (IQR 0.5–1.9). No patients developed flare during therapy. During FU period, 4 patients flared(3 Li and mixed LS and 1 SqM; 3 females) after discontinued therapy after completed 36 mo and in remission. Median duration to flare was 4.7 mo (range 0.99–10.1). This gives the flare incidence 0.0034 person-months. Cumulative probability risk of recurrence is estimated from our cohort that about 25% and 50% of flare within 46 and 54 mo, respectively. Over the study period(up to 78 mo), the probability of flare does not reach 75%. There was no major complication noted.

Conclusion:

The uniform treatment protocol given to all LS patients who required systemic therapy in our center enables us to assess the longterm outcome of the regimen more accurately. The therapy is effective in controlling the disease, however, after discontinuing therapy, 10% flare within 6 months. We proposed to continue the therapy longer, at least 48–54 months in order to decrease the rate of flare.

To cite this abstract, please use the following information:
Arkachaisri, Thaschawee, Torok, Kathryn S.; Localized Scleroderma Therapy: When Would Be a Good Time To Stop? [abstract]. Arthritis Rheum 2010;62 Suppl 10 :1683
DOI: 10.1002/art.29448

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