Liver Transplantation for Acute Intermittent Porphyria.

Lissing, Mattias; Nowak, Greg; Adam, René; Karam, Vincent; Boyd, Alexander; Gouya, Laurent; Meersseman, Wouter; Melum, Espen; Ołdakowska-Jedynak, Urszula; Reiter, Florian P; Colmenero, Jordi; Sanchez, Rosario; Herden, Uta; Langendonk, Janneke; Ventura, Paolo; Isoniemi, Helena; Boillot, Olivier; Braun, Felix; Perrodin, Stéphanie; Mowlem, Elizabeth; ... (2021). Liver Transplantation for Acute Intermittent Porphyria. Liver transplantation, 27(4), pp. 491-501. Wiley 10.1002/lt.25959

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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 cure but published data on outcome after LT are limited. We aimed to assess the pre-transplant characteristics, complications and outcomes for patients transplanted for AIP. Data was collected retrospectively from the European Liver Transplant Registry (ELTR) and from questionnaires sent to identified transplant and porphyria centers. We studied 38 patients transplanted in 12 countries 2002-2019. Median age at LT was 37 years (range 18-58) and 34 (89%) were female. Two patients were re-transplanted and nine died during follow-up. The 1-year and 5-year overall survival was 92% and 82%, which is comparable to other metabolic diseases transplanted during the same period. Advanced pretransplant neurological impairment was identified as a risk factor for mortality. The 5-year survival 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 had no significant effect on survival with a 5-year survival of 81% among 18 patients with a pretransplant GFR >60ml/min, and 71% among 14 patients with a pretransplant GFR <60ml/min (p=0.16). While 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. Liver transplantation 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 outcome. If other treatment options fail, evaluation for liver transplantation should be performed early.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Gastro-intestinal, Liver and Lung Disorders (DMLL) > Clinic of Visceral Surgery and Medicine

UniBE Contributor:

Perrodin, Stéphanie Fabienne

Subjects:

600 Technology > 610 Medicine & health

ISSN:

1527-6465

Publisher:

Wiley

Language:

English

Submitter:

Rahel Fuhrer

Date Deposited:

23 Dec 2020 12:16

Last Modified:

23 Apr 2021 01:32

Publisher DOI:

10.1002/lt.25959

PubMed ID:

33259654

Uncontrolled Keywords:

ELTR inherited metabolic disease neuropathy rare disease

BORIS DOI:

10.48350/150057

URI:

https://boris.unibe.ch/id/eprint/150057

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