Deferasirox for porphyria cutanea tarda: a pilot study

Area of Science:

• Dermatology
• Hepatology
• Internal Medicine

Background:

• Porphyria cutanea tarda (PCT) is the most common porphyria and can be challenging to treat.
• Iron overload is a contributing factor in PCT.
• Deferasirox is an oral iron-chelating agent approved for managing chronic iron overload.

Purpose of the Study:

• To evaluate the efficacy and safety of deferasirox in patients with PCT.
• To assess the impact of deferasirox on cutaneous manifestations and biochemical markers of PCT.

Main Methods:

• A prospective, open-label, 6-month pilot study was conducted.
• Ten patients with PCT were enrolled and treated with deferasirox (250-500 mg/d).
• Outcomes included changes in blister count, urinary porphyrin levels, and ferritin levels.

Main Results:

• Eight of ten patients completed the study.
• Seven patients experienced resolution of blistering.
• Six patients showed reduced urinary porphyrin levels, and seven had reduced ferritin levels.
• The treatment was generally well-tolerated.

Conclusions:

• Deferasirox demonstrated potential as a treatment for PCT, improving skin symptoms and reducing key biochemical markers.
• The drug was well-tolerated in this pilot study.
• Larger, controlled trials are warranted to confirm these findings and establish deferasirox as an alternative PCT treatment.