Deferasirox for porphyria cutanea tarda: A pilot study

Amit G. Pandya, Kaveh A. Nezafati, Mia Ashe-Randolph, Radha Yalamanchili

Research output: Contribution to journalArticle

7 Scopus citations

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

Original languageEnglish (US)
Pages (from-to)898-901
Number of pages4
JournalArchives of Dermatology
Volume148
Issue number8
DOIs
StatePublished - Aug 1 2012

ASJC Scopus subject areas

  • Dermatology

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    Pandya, A. G., Nezafati, K. A., Ashe-Randolph, M., & Yalamanchili, R. (2012). Deferasirox for porphyria cutanea tarda: A pilot study. Archives of Dermatology, 148(8), 898-901. https://doi.org/10.1001/archdermatol.2012.807