Pirfenidone gel in patients with localized scleroderma: a phase II study

Area of Science:

• Dermatology
• Rheumatology
• Pharmacology

Background:

• Localized scleroderma is an inflammatory and fibrotic skin condition primarily driven by transforming growth factor β.
• Current treatments for localized scleroderma lack standardization.
• Transforming growth factor β plays a key role in the pathogenesis of localized scleroderma.

Purpose of the Study:

• To evaluate the efficacy and safety of 8% pirfenidone gel for treating localized scleroderma.
• To assess the impact of pirfenidone gel on clinical, durometer, and histological measures of localized scleroderma.

Main Methods:

• An open-label, phase II clinical trial involving 12 patients with localized scleroderma.
• Patients received 8% pirfenidone gel application three times daily for six months.
• Clinical assessment included the modified Localized Scleroderma Skin Severity Index (mLoSSI), durometer measurements, and histological analysis (hematoxylin and eosin, Masson's trichrome stains).

Main Results:

• Significant reduction in mLoSSI scores from 5.83 to 0.83 (P=.002) after six months of treatment.
• A trend towards decreased plaque induration was observed (35.79 to 32.47, P=.05).
• Histological analysis revealed improvements in epidermal atrophy, inflammation, fibrosis, and annex atrophy (12.25 to 9.75, P=.032).

Conclusions:

• Topical pirfenidone gel demonstrates effectiveness in treating localized scleroderma, addressing both inflammatory and fibrotic aspects.
• The treatment exhibited a favorable safety profile with no detected side effects.
• Pirfenidone gel presents a viable, topically administered treatment option for localized scleroderma due to its efficacy and safety.