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.

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.