A Prospective Observational Study of Mycophenolate Mofetil Treatment in Progressive Diffuse Cutaneous Systemic Sclerosis of Recent Onset
The Journal of Rheumatology,

Mendoza FA et al. – Patients with diffuse progressive cutaneous systemic sclerosis (SSc) of recent onset treated with mycophenolate mofetil (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.

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.
  • Realtime PCR of skin biopsies demonstrated a marked decrease in expression of fibrosis–related genes.

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