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Real-world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study.
ESC Heart Fail
November 2024
Faculty of Medicine and University Hospital Cologne, Clinic III for Internal Medicine, University of Cologne, Cologne, Germany.
Aims: Data on the clinical profiles of patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) in the post-approval era of tafamidis 61 mg are lacking. Study aims were characterization of contemporary ATTR-CM patients, analysis of potential eligibility for the 'Transthyretin Amyloidosis Cardiomyopathy Clinical Trial' (ATTR-ACT) and identification of factors associated with the decision on tafamidis 61 mg treatment.
Methods And Results: This retrospective study analysed ATTR-CM patients seen at eight University Hospitals in the first year after approval of tafamidis 61 mg for ATTR-CM in Germany (April 2020 to March 2021).
Clinical outcomes for 2788 patients with transthyretin amyloidosis: Tafamidis meglumine early access program in France.
Arch Cardiovasc Dis
October 2024
Cardiology Department and French National Reference Centre for Cardiac Amyloidosis, Henri-Mondor University Hospital, AP-HP, GRC Amyloid Research Institute and IMRB, Inserm, Université Paris Est Créteil, 94010 Créteil, France. Electronic address:
Background: Early access experience in France with tafamidis meglumine, a selective transthyretin stabilizer for transthyretin-related amyloidosis cardiomyopathy (ATTR-CM), following transthyretin-related amyloidosis (ATTR) polyneuropathy approval and positive ATTR-ACT study results.
Aim: To describe the characteristics and clinical outcomes for patients in the French ATTR-CM tafamidis meglumine early access programme (28 Nov 2018 to 01 Jun 2021).
Methods: Patients with confirmed ATTR-CM received tafamidis meglumine 20mg/day or 80mg/day.
The effect of long-term tafamidis treatment on quality of life in people with transthyretin amyloid cardiomyopathy (ATTR-CM): A plain language summary.
Future Cardiol
October 2024
Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland, OH, USA.
Article Synopsis
- The ATTR-ACT study aimed to assess the effectiveness of tafamidis in treating transthyretin amyloid cardiomyopathy (ATTR-CM) over a period of up to 2.5 years, with participants either receiving tafamidis or a placebo.
- In the long-term extension study, participants who initially received tafamidis maintained their quality of life and heart failure symptoms, while those who switched from placebo to tafamidis experienced slower worsening of their conditions.
- The researchers concluded that tafamidis is beneficial in slowing down the decline in quality of life and heart failure symptoms for people with ATTR-CM, suggesting that early treatment is important.
Atrial Fibrillation as a Prognostic Factor for All-Cause Mortality in Patients With Transthyretin Amyloid Cardiomyopathy.
JACC CardioOncol
August 2024
Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV and Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain.
Background: Atrial fibrillation/atrial flutter (AF/AFL) are common manifestations of transthyretin amyloid cardiomyopathy (ATTR-CM) but have not been found to be predictive of mortality.
Objectives: This analysis aimed to examine whether baseline or historical AF/AFL at enrollment was prognostic for all-cause mortality.
Methods: In the ATTR-ACT (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial), a 30-month study of tafamidis vs placebo for ATTR-CM, AF/AFL was evaluated as an independent prognostic factor for all-cause mortality using Cox proportional hazards modelling.
Tafamidis therapy in transthyretin amyloid cardiomyopathy: a narrative review from clinical trials and real-world evidence.
Egypt Heart J
July 2024
Barking, Havering and Redbridge NHS Trust, London, UK.
Background: Amyloidosis is a heterogeneous group of disorders caused by the extracellular deposition of insoluble misfolded proteins, leading to end-organ damage. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a subtype in which a protein known as transthyretin accumulates within the heart tissue, progressively resulting in restrictive cardiomyopathy and heart failure. Due to the progressive nature of ATTR-CM, clinical management requires efficacious regimens to manage the debilitating condition and Tafamidis shows promising results in this regard.
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