A prospective open-label study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis.
OBJECTIVE
To evaluate the efficacy and safety of mycophenolate mofetil for the treatment of SSc.


METHODS
We recruited 15 patients with dcSSc to take part in an open-label study using mycophenolate mofetil to treat their disease over a 12-month period. The primary outcome measure was the modified Rodnan skin score (mRSS), whereas secondary outcomes included the Medsger severity score, pulmonary function studies, 2D echocardiograms and the Short Form Health Survey (SF)-36 questionnaire.


RESULTS
The mRSS significantly improved in those patients who tolerated the medication for >3 months (P < 0.0001), and there was a statistically significant improvement in the Medsger severity scores of the general (P = 0.05), peripheral vascular involvement (P = 0.05) and skin (P = 0.0003) scores. The SF-36 scores improved (P = 0.05) and the pulmonary function studies showed a trend towards improvement, though not of statistical significance. The mean pulmonary artery pressure by 2D echocardiography did not change.


CONCLUSIONS
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. Controlled trials are needed to further investigate this trend for improved pulmonary function studies.
Division of Rheumatology, Thomas Jefferson University, Philadelphia, PA 19107, USA. chris.derk@jefferson.edu
Chris T
CT
Derk
Elizabeth
E
Grace
Max
M
Shenin
Manisha
M
Naik
Steffan
S
Schulz
Wen
W
Xiong
D000328:Adult; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011446:Prospective Studies; D012129:Respiratory Function Tests; D045743:Scleroderma, Diffuse / Q000188:drug therapy*; D012720:Severity of Illness Index; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid