Document Detail

A prospective observational study of mycophenolate mofetil treatment in progressive diffuse cutaneous systemic sclerosis of recent onset.
MedLine Citation:
PMID:  22467932     Owner:  NLM     Status:  MEDLINE    
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.
CONCLUSION: 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.
Fabian A Mendoza; Sarah J Nagle; Jason B Lee; Sergio A Jimenez
Publication Detail:
Type:  Clinical Trial; Journal Article     Date:  2012-04-01
Journal Detail:
Title:  The Journal of rheumatology     Volume:  39     ISSN:  0315-162X     ISO Abbreviation:  J. Rheumatol.     Publication Date:  2012 Jun 
Date Detail:
Created Date:  2012-06-04     Completed Date:  2012-10-02     Revised Date:  2013-05-29    
Medline Journal Info:
Nlm Unique ID:  7501984     Medline TA:  J Rheumatol     Country:  Canada    
Other Details:
Languages:  eng     Pagination:  1241-7     Citation Subset:  IM    
Jefferson Institute of Molecular Medicine, and Scleroderma Center, Philadelphia, PA, USA.
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MeSH Terms
Disease Progression
Fibrillar Collagens / genetics,  metabolism
Fibrosis / drug therapy,  genetics,  pathology
Gene Expression / drug effects
Immunosuppressive Agents / therapeutic use*
Lung / drug effects,  pathology,  physiopathology
Middle Aged
Mycophenolic Acid / analogs & derivatives*,  therapeutic use
Prospective Studies
Respiratory Function Tests
Scleroderma, Diffuse / diagnosis*,  drug therapy*,  physiopathology
Skin / drug effects,  metabolism,  pathology
Time Factors
Treatment Outcome
Reg. No./Substance:
0/Fibrillar Collagens; 0/Immunosuppressive Agents; 24280-93-1/Mycophenolic Acid; 9242ECW6R0/mycophenolate mofetil

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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