Liver Transplantation for Acute Intermittent Porphyria

Mattias Lissing*, Greg Nowak, European Liver and Intestine Transplant Association, René Adam, Vincent Karam, Alexander Boyd, Laurent Gouya, Wouter Meersseman, Espen Melum, Urszula Ołdakowska-Jedynak, Florian P. Reiter, Jordi Colmenero, Rosario Sanchez, Uta Herden, Janneke Langendonk, Paolo Ventura, Helena Isoniemi, Olivier Boillot, Felix Braun, Stéphanie PerrodinElizabeth Mowlem, Staffan Wahlin

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Recurrent attacks of acute intermittent porphyria (AIP) result in poor quality of life and significant risks of morbidity and mortality. Liver transplantation (LT) offers a cure, but published data on outcomes after LT are limited. We assessed the pretransplant characteristics, complications, and outcomes for patients with AIP who received a transplant. Data were collected retrospectively from the European Liver Transplant Registry and from questionnaires sent to identified transplant and porphyria centers. We studied 38 patients who received transplants in 12 countries from 2002 to 2019. Median age at LT was 37 years (range, 18-58), and 34 (89%) of the patients were women. A total of 9 patients died during follow-up, and 2 patients were retransplanted. The 1-year and 5-year overall survival rates were 92% and 82%, which are comparable with other metabolic diseases transplanted during the same period. Advanced pretransplant neurological impairment was associated with increased mortality. The 5-year survival rate was 94% among 19 patients with moderate or no neuropathy at LT and 83% among 10 patients with severe neuropathy (P = 0.04). Pretransplant renal impairment was common. A total of 19 (51%) patients had a GFR < 60 mL/minute. Although few patients improved their renal function after LT, neurological impairments improved, and no worsening of neurological symptoms was recorded. No patient had AIP attacks after LT, except for a patient who received an auxiliary graft. LT is a curative treatment option for patients with recurrent attacks of AIP. Severe neuropathy and impaired renal function are common and increase the risk for poor outcomes. If other treatment options fail, an evaluation for LT should be performed early.

Original languageEnglish
Pages (from-to)491-501
Number of pages11
JournalLiver Transplantation
Issue number4
Publication statusPublished - Apr 2021

Bibliographical note

We thank Penelope Stein, King's College Hospital, London; Bridget Gunson, University Hospitals Birmingham; Jean Charles Deybach, University Denis Diderot, Paris; Gerald Denk, University Hospital, Ludwig Maximilian University (LMU) Munich; David Cassiman, University of Leuven; Eliane Sardh and Daphne Vassiliou, Porphyria Centre Sweden; and research coordinator Shyhanaz Alibaks, Erasmus Medical Center (MC), University Medical Center, Rotterdam for valuable contributions in the collection of data. The Organ Sharing Organizations, the French Agence de la Biom?decine (ABM) (Sami Djabbour), the Dutch transplant foundation (NTS) (Maaike de Wolf), the Eurotransplant Foundation (Marieke Van Meel), the Spanish Liver Transplant Registry (RETH) (Gloria de la Rosa), the UK?Ireland National Healt Services Blood and Transplant (NHSBT) (Michael Daynes), and the Scandiatransplant (Ilse Duus Weinreich), are acknowledged for the data sharing and cross-check. The ELTR has received logistic support from the Paul Brousse Hospital (Assistance Publique-Hôpitaux de Paris).

Publisher Copyright:
Copyright © 2020 The Authors. Liver Transplantation published by Wiley Periodicals LLC on behalf of American Association for the Study of Liver Diseases.


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