TY - JOUR
T1 - Deferasirox for porphyria cutanea tarda
T2 - A pilot study
AU - Pandya, Amit G.
AU - Nezafati, Kaveh A.
AU - Ashe-Randolph, Mia
AU - Yalamanchili, Radha
PY - 2012/8
Y1 - 2012/8
N2 - 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
AB - 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
UR - http://www.scopus.com/inward/record.url?scp=84865701058&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84865701058&partnerID=8YFLogxK
U2 - 10.1001/archdermatol.2012.807
DO - 10.1001/archdermatol.2012.807
M3 - Article
C2 - 22911183
AN - SCOPUS:84865701058
SN - 0003-987X
VL - 148
SP - 898
EP - 901
JO - Archives of Dermatology
JF - Archives of Dermatology
IS - 8
ER -