Placebo-controlled Study of Methotrexate in Juvenile Localised Scleroderma PDF Print E-mail
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.
 
More articles :

» Biomarkers Predict Lung Decline in Systemic Sclerosis

Predicting risk of pulmonary fibrosis and pulmonary hypertension is an urgent priority in systemic sclerosis. Lung function often declines rapidly in the first years after diagnosis, and interstitial lung disease (ILD), although highly variable in...

» Gene Linked to Autoimmune Diseases

Differences in the sequence of a single gene may be partly responsible for causing around 2% of relatively common autoimmune disorders including diabetes and arthritis.The gene codes for an enzyme called sialic acid acetylesterase (SIAE) that...

» Methotrexate Effective In Juvenile Localized Scleroderma

is effective in when given with a short course of steroids, an Italian randomized study confirmed.Among children ages 6 to 17 receiving methotrexate, 67.4% completed the yearlong trial without flaring, compared with 29.2% of those given placebo...

» A System Out of Breath: How Hypoxia Possibly Contributes to the Pathogenesis of Systemic Sclerosis

T. W. van Hal, L. van Bon, and T. R. D. J. RadstakeReceived 20 May 2011; Revised 18 August 2011; Accepted 7 September 2011Systemic sclerosis (SSc) is typified by vascular alterations and immunological disturbances and fibrosis of the skin and...

» NeoStem Awarded Grant To Develop New Treatment for Skin Wounds In Scleroderma

NeoStem, as a leader in the emerging cellular therapy industry, announced that it had received an award under the Small Business Innovative Research Program of $147,765 for the “Development of Adult Pluripotent Very Small Embryonic Like (VSEL)...

» Millions Around World To Observe Rare Disease Day

Millions Around World To Observe Rare Disease DayThe Relief Foundation will be joining the National Organization for Rare Disorders (NORD) and others around the world in observing World Rare Disease Day on February 28, 2011.  On this day,...