Liver transplantation for transthyretin amyloidosis: experience of a single center in Italy

Berardi, Sonia (2008) Liver transplantation for transthyretin amyloidosis: experience of a single center in Italy, [Dissertation thesis], Alma Mater Studiorum Università di Bologna. Dottorato di ricerca in Trapianto di fegato: immunologia clinica e di base ed immunodepressione, 20 Ciclo. DOI 10.6092/unibo/amsdottorato/1128.
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Liver transplantation is the only definitive treatment for transthyretin amyloidosis, with an excellent 5-year survival in endemic countries where the Met30 mutation is predominant. We report our experience of liver transplantation for transthyretin amyloidosis. We reviewed the clinical records of 17 transplanted patients (11 males, 6 females; age at liver transplant: 45.7±11.7 years). We had a wide spectrum of non-Met30 mutations (52.9%), with a predominance of Gln89 (23.5%). Five-year survival after transplantation was 43.8%; at multivariate analysis, both non-Met30 mutations (HR 17.3, 95% CI 1.03-291.7) and modified BMI (HR 0.50, 95% CI 0.29-0.87) showed significant and independent prognostic roles (P=0.048 and P=0.015, respectively). Five out of the 9 non-Met30 carriers received combined heart transplantation because of severe cardiomyopathy; they showed a trend towards a better prognosis vs. the 4 patients who did not receive combined heart transplantation (although not statistically significant; P=0.095). At follow-up, no significant improvement of transthyretin amyloidosis manifestations was observed. The results of liver transplantation for transthyretin amyloidosis in our population are poorer than those reported in the literature probably because of the high prevalence of non-Met30 mutations.

Tipologia del documento
Tesi di dottorato
Berardi, Sonia
Dottorato di ricerca
Settore disciplinare
Settore concorsuale
Parole chiave
peripheral neuropathy heart transplantation
Data di discussione
23 Giugno 2008

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