|Placebo-controlled Study of Methotrexate in Juvenile Localised Scleroderma|
|Wednesday, 09 February 2011 16:24|
According to the results of this placebo-controlled study, methotrexate is an effective treatment for juvenile localised scleroderma (JLS).
The authors note that JLS is a chronic progressive fibrotic process of the skin causing permanent disability and aesthetic damage. There is deep tissue involvement, and around 20% will have another organ involved. Although it is the most common form of scleroderma in children, it remains poorly understood. There is no universally accepted treatment for this condition; however methotrexate (MTX) has been reported to be beneficial.
In this study, they randomised 70 patients aged 6-17 years with active JLS to receive double-blind treatment with methotrexate (15mg/m2; max 20mg; n=46) or placebo (n=24) once weekly, alongside oral prednisolone (1mg/kg/day; max 50mg) for the initial three months (followed by one month of dose tapering). Treatment was continued for 12 months, or until treatment failure, if sooner. Patients were assessed during six clinical visits throughout the treatment period. The primary endpoint was the rate of response to treatment at the end of the 12 month period. Response was defined as no new lesions, a skin score rate (SSR) score of a target lesion <1, and a decrease in lesion temperature by at least 10% compared to baseline.
Around half of the enrolled patients completed the 12-month study, including 67.4% of the MTX group and 29.2% of the placebo group (premature discontinuation due to disease flare in all cases). All 70 enrolled patients were included in the intention-to-treat analysis of efficacy. The main findings were as follows:
• At the final visit, 67.4% of the MTX group and 29.2% of the placebo-treated patients had responded to treatment (p=0.001).
• After an initial response, disease relapsed in 15 MTX patients (32.6%) and 17 placebo (70.8%) (p<0.005).
• The likelihood to experience a flare of disease in the MTX group was around one third of that in the placebo group (hazard ratio [HR] 2.82, 95% CI 1.40 - 5.66).
• New lesions appeared in three MTX patients (6.5%) vs. four of those on placebo (16.7%), mean SSR decreased from 1 to 0.79 with MTX vs. 1.1 on placebo (p=0.011), and the mean target lesion temperature decreased by 44.4% vs. 12.1%, respectively (p=0.024)
The authors conclude that methotrexate in combination with a short course of prednisone is beneficial and well tolerated as a treatment for localised scleroderma in children.
Source: Pocock, N. (2011), "Placebo-controlled Study of Methotrexate in Juvenile Localised Scleroderma"; taken from original article here.