title: Pirfenidone gel in patients with localized scleroderma: a phase II study.
abstract: BACKGROUND
  Localized scleroderma is an inflammatory disease in its first stages and a fibrotic process in later stages, principally mediated by the transforming growth factor β. To date, there is no standard treatment. The objective of this study was to determine the effectiveness and safety of 8% pirfenidone gel in patients with localized scleroderma.
  
  
  METHODS
  This was an open phase II clinical trial that included 12 patients. Treatment with pirfenidone was indicated, three times daily for 6 months. Patients were evaluated clinically with the modified Localized Scleroderma Skin Severity Index (mLoSSI), as well with a durometer and histologically using hematoxylin and eosin stain and Masson's trichrome stain.
  
  
  RESULTS
  The baseline mLoSSI average scores were 5.83 ± 4.80 vs. 0.83 ± 1.75 (P = 0.002) at 6 months. The initial durometer induration of the scleroderma plaques was 35.79 ± 9.10 vs. 32.47 ± 8.97 at 6 months (P = 0.05). We observed histopathological improvement with respect to epidermal atrophy, inflammation, dermal or adipose tissue fibrosis and annex atrophy from 12.25 ± 3.25 to 9.75 ± 4.35 (P = 0.032). The 8% pirfenidone gel application was well tolerated, and no side effects were detected.
  
  
  CONCLUSIONS
  This is the first study on the therapeutic use of pirfenidone gel in localized scleroderma. It acts on both the inflammatory and the fibrotic phases. Considering its effectiveness, good safety profile and the advantage of topical application, pirfenidone is a treatment option in this condition.
authors.forename: Marco
authors.initials: M
authors.lastname: Rodríguez-Castellanos
authors.forename: Alberto
authors.initials: A
authors.lastname: Tlacuilo-Parra
authors.forename: Sergio
authors.initials: S
authors.lastname: Sánchez-Enríquez
authors.forename: Ezequiel
authors.initials: E
authors.lastname: Vélez-Gómez
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Guevara-Gutiérrez
mesh_terms: D000287:Administration, Topical; D000328:Adult; D000700:Analgesics / Q000008:administration & dosage; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000008:administration & dosage*; D005260:Female; D005782:Gels; D006801:Humans; D008297:Male; D008875:Middle Aged; D011728:Pyridones / Q000008:administration & dosage*; D012594:Scleroderma, Localized / Q000175:diagnosis* / Q000188:drug therapy*
chemical_list: D000700:Analgesics; D000894:Anti-Inflammatory Agents, Non-Steroidal; D005782:Gels; D011728:Pyridones; C093844:pirfenidone