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Sequential liver and bone marrow transplantation for treatment of erythropoietic protoporphyria.

Rand EB, Bunin N, Cochran W, Ruchelli E, Olthoff KM, Bloomer JR

Department of Pediatrics, University of Pennsylvania, Children's Hospital of Philadelphia, 324 S 34th St, Philadelphia, PA 19104, USA. rand@email.chop.edu

Erythropoietic protoporphyria is a disorder of heme synthesis in which deficient ferrochelatase activity leads to excess production and biliary excretion of protoporphyrin. The main clinical features, photosensitivity and hepatobiliary disease that may progress to liver failure, are caused by the toxicity of protoporphyrin. Liver transplantation has been used to treat liver failure in erythropoietic protoporphyria, but excess production of protoporphyrin by the bone marrow continues causing recurrence of liver disease in the majority of patients. This is the first report of successful sequential liver and bone marrow transplantation in a patient with liver failure as a result of erythropoietic protoporphyria. This combination corrected the severe phenotype, resolving the severe photosensitivity and halting erythropoietic protoporphyria associated liver graft injury. Splenectomy seemed to facilitate the successful bone marrow transplant.

Published 4 December 2006 in Pediatrics, 118(6): e1896-9.
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