title: A prospective observational study of mycophenolate mofetil treatment in progressive diffuse cutaneous systemic sclerosis of recent onset.
abstract: OBJECTIVE
  A prospective observational study of mycophenolate mofetil (MMF) treatment in patients with diffuse progressive cutaneous systemic sclerosis (SSc) of recent onset.
  
  
  METHODS
  Twenty-five previously untreated consecutive patients with recent-onset (< 24 mo) diffuse progressive cutaneous SSc received MMF as the only disease-modifying therapy. Modified Rodnan skin score (mRSS) and affected body surface area (BSA) were compared from initiation of MMF to study end. Pulmonary function tests performed at the same institution before therapy and at study end were available in 15 patients. Histopathology and real-time PCR assessment of fibrosis-related gene expression were performed before and after treatment in skin biopsies from 3 patients.
  
  
  RESULTS
  At 18.2 ± 8.73 months of MMF therapy (median 2000 mg/day) the mRSS decreased from 24.56 ± 8.62 to 14.52 ± 10.9 (p = 0.0004) and the affected BSA from 36% ± 16% to 14% ± 13.3% (p = 0.00001). Pulmonary function tests remained stable from initiation of MMF to the end of the study. Skin histopathology showed a remarkable reduction in accumulation of fibrotic tissue. Real-time PCR of skin biopsies demonstrated a marked decrease in expression of fibrosis-related genes.
  
  
  CONCLUSIONS
  Patients with diffuse progressive cutaneous SSc of recent onset treated with MMF experienced marked improvement in skin involvement and stabilization of pulmonary function. Skin biopsies from 3 patients demonstrated histopathological improvement and decreased expression of fibrosis-related genes.
authors.affiliation: Jefferson Institute of Molecular Medicine, and Scleroderma Center, Philadelphia, PA, USA.
authors.forename: Fabian A
authors.initials: FA
authors.lastname: Mendoza
authors.forename: Sarah J
authors.initials: SJ
authors.lastname: Nagle
authors.forename: Jason B
authors.initials: JB
authors.lastname: Lee
authors.forename: Sergio A
authors.initials: SA
authors.lastname: Jimenez
mesh_terms: D018450:Disease Progression; D024022:Fibrillar Collagens / Q000235:genetics / Q000378:metabolism; D005355:Fibrosis / Q000188:drug therapy / Q000235:genetics / Q000473:pathology; D015870:Gene Expression / Q000187:drug effects; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008168:Lung / Q000187:drug effects / Q000473:pathology / Q000503:physiopathology; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011379:Prognosis; D011446:Prospective Studies; D012129:Respiratory Function Tests; D045743:Scleroderma, Diffuse / Q000175:diagnosis* / Q000188:drug therapy* / Q000503:physiopathology; D012867:Skin / Q000187:drug effects / Q000378:metabolism / Q000473:pathology; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D024022:Fibrillar Collagens; D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid