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dc.contributor.authorLissing, Mattias
dc.contributor.authorNowak, Greg
dc.contributor.authorAdam, René
dc.contributor.authorKaram, Vincent
dc.contributor.authorBoyd, Alexander
dc.contributor.authorGouya, Laurent
dc.contributor.authorMeersseman, Wouter
dc.contributor.authorMelum, Espen
dc.contributor.authorOłdakowska‐Jedynak, Urszula
dc.contributor.authorReiter, Florian P.
dc.contributor.authorColmenero, Jordi
dc.contributor.authorSanchez, Rosario
dc.contributor.authorHerden, Uta
dc.contributor.authorLangendonk, Janneke
dc.contributor.authorVentura, Paolo
dc.contributor.authorIsoniemi, Helena
dc.contributor.authorBoillot, Olivier
dc.contributor.authorBraun, Felix
dc.contributor.authorPerrodin, Stéphanie
dc.contributor.authorMowlem, Elizabeth
dc.contributor.authorWahlin, Staffan
dc.contributor.authorEuropean Liver and Intestine Transplant Association
dc.date.accessioned2021-04-21T05:13:33Z
dc.date.available2021-04-21T05:13:33Z
dc.date.issued2021-01-29
dc.date.submitted2020-05-21
dc.identifier.issn1527-6465
dc.identifier.issn1527-6473
dc.identifier.otherlt25959
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/321373
dc.descriptionFunder: Alnylam Pharmaceuticals; Id: http://dx.doi.org/10.13039/100006400
dc.description.abstractRecurrent 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.
dc.languageen
dc.subjectOriginal Article
dc.subjectOriginal Articles
dc.titleLiver Transplantation for Acute Intermittent Porphyria
dc.typeArticle
dc.date.updated2021-04-21T05:13:33Z
prism.endingPage501
prism.issueIdentifier4
prism.publicationNameLiver Transplantation
prism.startingPage491
prism.volume27
dc.identifier.doi10.17863/CAM.68494
dcterms.dateAccepted2020-11-03
rioxxterms.versionofrecord10.1002/lt.25959
rioxxterms.versionAO
rioxxterms.versionVoR
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.contributor.orcidPerrodin, Stéphanie [0000-0002-2767-3219]


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