To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria
Primary objective - the elimination of all blistering within 6 months of treatment.
Secondary objective - decrease in total body iron levels.
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.
- diagnosis of Porphyria Cutanea Tarda based on clinical exam and 24-hour urine
- 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
- 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.