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Retrospective follow-up of a cohort of TTR amyloidosis patients treated with Tafamidis at Montpellier University Hospital
Mémoire de Master / Thèse d'exercice   Open Access

Retrospective follow-up of a cohort of TTR amyloidosis patients treated with Tafamidis at Montpellier University Hospital

Johanna Krief
Masters , Université de Montpellier
09/06/2023

Résumé

TTR cardiac amyloidosis Tafamidis Cardiac biomarkers NT-proBNP Amylose cardiaque à TTR Fibrillation atriale Mortality Atrial fibrillation Mortalité
Transthyretin cardiac amyloidosis is an important cause of heart failure. Recently, Tafamidis, a specific treatment for cardiac amyloidosis, has shown efficacy on mortality. The purpose of this study was to describe survival to death or hospitalization for cardiovascular events in patients treated by Tafamidis and determine predictors of survival of these patients. Materials and methods: we retrospectively included consecutive patients with a new diagnosis of ATTR-CM with an indication for Tafamidis at the University Hospital of Montpellier from August 27, 2012, and January 10, 2023. We collected survival data, but also clinical, biological, ultrasound and therapeutic factors that may have an impact on survival. Results: a total of 128 patients were included. During a median follow-up of 15 months, 42 patients (33%) died or were hospitalized for cardiovascular causes with a median survival of 35 months for these parameters. Several prognostic factors were identified: old age at diagnosis is a protective factor [hazard ratio (HR) 0.93, 95% confidence interval (CI) 0.89-0.97, p < 0.01] and obesity is a risk factor [HR 3.95, 95% CI 1.45-10.76, p = 0.01]. More importantly, NT-proBNP >1000 ng/mL at diagnosis is a major prognostic factor [HR 4.10, 95% CI 1.64-10.25, p < 0.01]. Discussion: ATTR-CM patients on Tafamidis have a poor prognosis in the short term, despite the use of Tafamidis. Prompt initiation of therapy is necessary, and evaluation of important prognostic factors such as NT-proBNP are a major issue in this condition.

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