Erythropoietin for the Treatment of Porphyria Cutanea Tarda in End Stage Renal Disease


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Porphyria Cutanea Tarda (PCT) is an iron-related disease that results from acquired inhibition of hepatic uroporphyrinogen decarboxylase (UROD), the fifth enzyme in the heme biosynthetic pathway. Clinically, PCT presents with chronic photosensitive vesiculo-bullous skin lesions that are painful and prone to infection and scarring. Renal disease is often associated with iron overload, and is a risk factor for the development of PCT. It is difficult to treat PCT associated with renal disease due to the presence of anemia, which prevents treatment of these patients by phlebotomy. Anemia in these patients results largely from decreased production of erythropoietin by the diseased kidneys. Erythropoietin administration can mobilize excess iron, correct anemia and support phlebotomy in these patients. This study will analyze data from an unblinded trial of 6 patients who were treated with recombinant human erythropoietin for their renal disease-associated PCT. The aims are to determine the safety and efficacy of erythropoietin treatment of PCT in patients with end stage renal disease as assessed by improvements in plasma porphyrins, measures of iron status, clinical improvement and side effects, and to contribute to developing clear guidelines for erythropoietin treatment of these patients.



Porphyria catania tarda End stage renal disease