Deferasirox for porphyria cutanea tarda: a pilot study.
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.


METHODS
Prospective, open-label, noncomparative study.


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


METHODS
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.


METHODS
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.


BACKGROUND
clinicaltrials.gov Identifier: NCT00599326.
Department of Dermatology, University of Texas Southwestern Medical Center, Dallas, TX 75390-9190, USA. amit.pandya@utsouthwestern.edu
Amit G
AG
Pandya
Kaveh A
KA
Nezafati
Mia
M
Ashe-Randolph
Radha
R
Yalamanchili
D001565:Benzoates / Q000009:adverse effects / Q000627:therapeutic use*; D001768:Blister / Q000188:drug therapy*; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D017119:Porphyria Cutanea Tarda / Q000097:blood / Q000188:drug therapy* / Q000652:urine; D011166:Porphyrins / Q000652:urine; D016896:Treatment Outcome; D014230:Triazoles / Q000009:adverse effects / Q000627:therapeutic use*
D001565:Benzoates; D007502:Iron Chelating Agents; D011166:Porphyrins; D014230:Triazoles; C545835:porphyrin 5,10,15-tris(N-methylpyridinium-4-yl)-20-(1-pyrenyl)-21H,23H-porphyrin; D005293:Ferritins; D000077588:Deferasirox