Pirfenidone Clinical Study 5

An open phase II clinical trial to determine the effectiveness and safety of 8% pirfenidone gel in patients with localized scleroderma.

• N= 12 patients with clinical and histopathological diagnosis of active localized scleroderma. 
• Patients were instructed to apply 8% pirfenidone gel three times daily for 6 months using the standard fingertip unit (0.5 g for an area of 100 to 120 cm2)
• Assessments performed were: 

• All patients showed a sustained improvement in their lesions throughout the study.
• The basal mLoSSI average was 5.83 ± 4.80 vs. 1.33 ± 1.92 at 3 months of treatment (P = 0.003) vs. 0.83 ± 1.75 at 6 months (P = 0.002). (Figure 10).
• The improvement in the localized scleroderma lesions with the application of 8% pirfenidone gel was evident with the mLoSSI score, showing an average improvement of 85.7% and 100% improvement in 66% of the cases at 6 months of treatment (P = 0.002).
• The mean induration of the localized scleroderma plaques diminished between the basal and final measurements (35.79 DU vs. 32.47 DU, P = 0.05), showing a tendency toward reduction. (Figure 11)
• Patients’ assessments of the hardness of the lesions and the presence of pruritus showed improvement with the application of pirfenidone, although the difference was not significant.
• Histopathological evaluations showed 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.

Considering its effectiveness, good safety profile, and the advantage of topical application, pirfenidone 8% gel is a treatment option in localized scleroderma.