Therapy of porphyria cutanea tarda

Abstract

Porphyria cutanea tarda (PCT) is the most common type of porphyria. There is an association of PCT with haemochromatosis, diabetes mellitus and hepatitis C infection. The basis of treatment of PCT consists of three elements: avoidance of triggering factors, iron depletion and porphyrin elimination. Alcohol and certain systemic medical drugs, such as oestrogens (or tuberculostatics), should be considered as triggering factors, and as far as possible, avoided. Other triggering factors, such as chronic haemodialysis in renal insufficiency, need a different approach. The hallmark in iron depletion is phlebotomy. Porphyrin elimination is achieved using low-dose chloroquin therapy. The treatment is safe and effective but has its limits in cases with haemochromatosis (HFE) gene mutations. Here iron depletion needs additional phlebotomy. In patients with chronic haemodialysis-associated PCT, chloroquine is ineffective. Erythropoietin, desferroxamine and small-volume phlebotomy have been employed to control the disease. Childhood PCT is very rare. No controlled studies are available, but published experience suggests that body weight-adapted chloroquine therapy or small volume phlebotomy might be useful.

Deferasirox (Exjade®) for the Treatment of Iron Overload

“…To date, the deferasirox clinical development program has focused on the treatment of patients with transfusional iron overload; however, a wider perspective is now being taken with the investigation of deferasirox in a number of other conditions including hereditary hemochromatosis (HH; characterized by progressive iron overloading through increased intestinal absorption [57]), chronic hepatitis C [58], porphyria cutanea tarda (a common type of porphyria which can be associated with hemochromatosis [59]), and mucormycosis.…”

Cutaneous porphyrias part II: treatment strategies

“…The safety and efficacy of low‐dose chloroquine (125 to 250 mg administered orally twice per week) and hydroxychloroquine (100 mg, orally, two or three times per week) are comparable with those of phlebotomy treatment in PCT, but these forms of therapy are much more convenient and less expensive and thus are preferred at some centers . Importantly, antimalarials do not cause significant iron depletion and may not be appropriate for PCT patients with significant iron overload . Likewise, patients with insufficient renal function to excrete the high levels of porphyrin complexes formed by antimalarial medications require alternative therapies (see Additional therapies for PCT ).…”

Stoffwechselerkrankungen der Leber

“…Vermutlich wirkt Chloroquin als Chelator für Porphyrine und fördert ihre Ausscheidung über die Niere[44]. Die Therapie wird mehrere Monate bis zu einem Jahr durchgeführt und führt in der Regel zu einer Normalisierung der erhöhten Prophyrinausscheidung sowie zu einer klinischen Remission[30]. Bei der Therapie der kutanen Effloreszenzen müssen zusätzlich Maßnahmen zum Schutz vor UV-Strahlen (schützende Kleidung und Kopfbedeckung, Sonnencreme mit hohem Lichtschutzfaktor) zur Anwendung kommen.…”