Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be inconvenient and cause anemia in some patients.

Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity for iron. The medication is administered orally, which if effective for PCT would make it a more convenient and possibly more tolerable option for patients.

• Deferasirox (Drug), also known as Exjade: 250 mg of deferasirox once daily for 6 months

Primary outcomes

• Number of Participants Showing Reduction or Elimination of Skin Blistering [Within 6 months of treatment.]: The present trial was undertaken to determine if oral deferasirox could be useful in the treatment of PCT. Monthly clinic visits with a physical examination was conducted to assess the skin for blisters.

Secondary outcomes

• Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [6 months]: Patients with PCT usually have normal or elevated serum iron and ferritin levels as well as increased iron absorption. Phlebotomy is conducted to analyzes the ferritin levels. Urine collection is performed and samples of the urine are analyzed for porphyrin levels.

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* diagnosis of Porphyria Cutanea Tarda based on clinical exam and 24-hour urine porphyrin levels
* have Porphyria Cutanea Tarda for at least 3 months prior to enrollment with active blistering (3 blisters or erosions per month)
* women of childbearing potential must use an effective method of contraception during the study, however this cannot include hormonal contraception (oral contraceptives, hormone patches, Depo-Provera injections, NUVA Ring, etc.)
* treatment naive patients or patients unresponsive or intolerant of phlebotomy
* Ferritin level is greater than or equal to 25ng/mL

Exclusion Criteria:

* patients with serum creatinine above the upper limit of normal
* patients receiving phlebotomy who are controlled on this therapy
* pregnant or breast feeding females
* patients with liver transaminases more than 5 times the upper limit of normal
* patients with a history of hypersensitivity to deferasirox
* patients with a history of pre-existing renal condition, or receiving medication that depresses renal function
* patients on other chelators
* history of non-compliance to medical regimens.

Participant flow

Outcome results

• Number of Participants Showing Reduction or Elimination of Skin Blistering (Primary), Number, participants: Deferasirox 0
• Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level (Secondary), Number, participants: Deferasirox 8

Adverse events

Other adverse events by term

Limitations and caveats: 10 patients were enrolled, of which 8 were compliant with therapy and had evaluable data. Of the 2 patients dropped from the study, one patient was lost to follow-up after 2 months and another was noncompliant with medication dosing.

• Results publication: Pandya AG, Nezafati KA, Ashe-Randolph M, Yalamanchili R. Deferasirox for porphyria cutanea tarda: a pilot study. Arch Dermatol. 2012 Aug;148(8):898-901. doi: 10.1001/archdermatol.2012.807. PMID 22911183

• Amit Pandya, M.D., Principal investigator, UT Southwestern Medical Center at Dallas - Department of Dermatology