Deferasirox for porphyria cutanea tarda: a pilot study.

Abstract

OBJECTIVE:

To determine the efficacy and safety of deferasirox (an oral iron-chelating agent approved to reduce iron stores in patients with chronic iron overload due to blood transfusions) in a pilot trial for the treatment of patients with porphyria cutanea tarda (PCT), the most common of the porphyrias and often difficult to treat.

DESIGN:

Prospective, open-label, noncomparative study.

SETTING:

University-affiliated tertiary health care center in Dallas, Texas.

PATIENTS:

Ten patients with PCT were enrolled in this 6-month study. The diagnosis was established by documenting the presence of elevated porphyrin level in the urine and a history of developing 3 or more blisters per month for at least 3 months prior to enrollment. Patients were treated with 250 mg/d of deferasirox, with an increase to 500 mg/d after 2 months if new blisters continued to develop.

MAIN OUTCOME MEASURE:

The improvement in number of blisters at the end of the 6-month treatment period was assessed.

RESULTS:

Of 10 patients, 8 completed the study. Seven had resolution of blistering, 6 had a reduction in urinary porphyrin levels, and 7 had a reduction in ferritin levels. The treatment was well tolerated.

CONCLUSIONS:

In this small pilot study, deferasirox induced improvement in cutaneous findings of PCT in 8 patients who completed 6 months of treatment. Most patients also had a substantial reduction in urinary porphyrin and ferritin levels. Future larger controlled studies are needed to confirm these findings. Deferasirox may be a useful alternative to existing treatment modalities for PCT.

TRIAL REGISTRATION:

clinicaltrials.gov Identifier: NCT00599326.

• Authors: Amit G Pandya, Kaveh A Nezafati, Mia Ashe-Randolph, Radha Yalamanchili
• Journal: Archives of dermatology (Arch Dermatol) Vol. 148 Issue 8 Pg. 898-901 (Aug 2012) ISSN: 1538-3652 [Electronic] United States
• PMID: 22911183 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Benzoates
• Iron Chelating Agents
• Porphyrins
• Triazoles
• porphyrin 5,10,15-tris(N-methylpyridinium-4-yl)-20-(1-pyrenyl)-21H,23H-porphyrin
• Ferritins
• Deferasirox

Topics

• Benzoates (adverse effects, therapeutic use)
• Blister (drug therapy)
• Deferasirox
• Female
• Ferritins (blood)
• Humans
• Iron Chelating Agents (adverse effects, therapeutic use)
• Male
• Middle Aged
• Pilot Projects
• Porphyria Cutanea Tarda (blood, drug therapy, urine)
• Porphyrins (urine)
• Treatment Outcome
• Triazoles (adverse effects, therapeutic use)