title: Liver transplantation for erythropoietic protoporphyria liver disease.
abstract: In erythropoietic protoporphyria (EPP), there is excessive production of protoporphyrin, primarily in the bone marrow, resulting in increased biliary excretion of this heme precursor. Some patients will develop progressive liver disease that may ultimately require liver transplantation. However, excessive production of protoporphyrin by the bone marrow continues after transplantation, which may cause recurrent disease in the allograft. This study was performed to define post-transplant survival, the risk of recurrent disease, and specific management issues in patients transplanted for EPP liver disease. The patients studied consisted of twelve males and eight females, with an average age of 31 (range, 13-56) years at the time of transplantation. The estimated maximum MELD score prior to transplant was 21 (range, 15-29). Unique complications in the perioperative period were light induced tissue damage in four patients and neuropathy in six, requiring prolonged mechanical ventilation in four. Patient and graft survival rates were 85% at 1 year, 69% at 5 years, and 47% at 10 years. Recurrent EPP liver disease occurred in 11 of 17 patients (65%) who survived more than 2 months. Three patients were retransplanted at 1.8, 12.6, and 14.5 years after the initial transplant for recurrent EPP liver disease. In conclusion, the 5-year patient survival rate in patients transplanted for EPP liver disease is good, but the recurrence of EPP liver disease appears to diminish long term graft and patient survival.
authors.affiliation: Department of Medicine, University of Alabama at Birmingham, 1530 Third Avenue South, Birmingham, AL 35294-0005, USA. bmcguire@uab.edu
authors.forename: Brendan M
authors.initials: BM
authors.lastname: McGuire
authors.forename: Herbert L
authors.initials: HL
authors.lastname: Bonkovsky
authors.forename: Robert L
authors.initials: RL
authors.lastname: Carithers
authors.forename: Raymond T
authors.initials: RT
authors.lastname: Chung
authors.forename: Leonard I
authors.initials: LI
authors.lastname: Goldstein
authors.forename: John R
authors.initials: JR
authors.lastname: Lake
authors.forename: Anna S
authors.initials: AS
authors.lastname: Lok
authors.forename: Carol J
authors.initials: CJ
authors.lastname: Potter
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Rand
authors.forename: Michael D
authors.initials: MD
authors.lastname: Voigt
authors.forename: Pamela R
authors.initials: PR
authors.lastname: Davis
authors.forename: Joseph R
authors.initials: JR
authors.lastname: Bloomer
mesh_terms: D000293:Adolescent; D000328:Adult; D015415:Biomarkers / Q000378:metabolism; D001853:Bone Marrow / Q000378:metabolism; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D006801:Humans; D016031:Liver Transplantation; D008297:Male; D008875:Middle Aged; D046351:Protoporphyria, Erythropoietic / Q000378:metabolism / Q000473:pathology / Q000601:surgery*; D011524:Protoporphyrins / Q000378:metabolism; D012008:Recurrence; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers; D011524:Protoporphyrins