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A prospective open-label study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis
Rheumatology, 10/23/09

Derk CT et al. – In this prospective open-label study of mycophenolate mofetil for the treatment of dcSSc, we observed significant improvements in skin scores, peripheral vascular involvement and patient-perceived health status. Pulmonary function studies did not worsen as expected, but instead showed a trend towards improvement.

• 15 patients with dcSSc
• Open-label study using mycophenolate mofetil to treat their disease over 12-month period
• Primary outcome measure was modified Rodnan skin score (mRSS), whereas secondary outcomes included Medsger severity score, pulmonary function studies, 2D echocardiograms and Short Form Health Survey (SF)-36 questionnaire

• mRSS significantly improved in patients who tolerated medication for > 3 months , and there was a statistically significant improvement in the Medsger severity scores of the general , peripheral vascular involvement and skin scores
• SF-36 scores improved and pulmonary function studies showed trend towards improvement, though not of statistical significance
• No change in mean pulmonary artery pressure by 2D echocardiography

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