{"id":"705520427f97","type":"article","url":"https://hartvaat.nl/2018/09/13/tafamidis-bij-transthyretine-amyloid-cardiomyopathie-nejm-attr-act/","title":"Tafamidis bij transthyretine amyloïd cardiomyopathie: NEJM ATTR-ACT","title_en":"Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy.","category":"hartfalen","category_label":"Hartfalen","professions":["cardioloog"],"tags":["cardiale-amyloidose","cardiomyopathie-gerichte-therapie"],"journal":"The New England journal of medicine","doi":"10.1056/NEJMoa1805689","source_url":"https://doi.org/10.1056/NEJMoa1805689","authors":["Mathew S Maurer","Jeffrey H Schwartz","Balarama Gundapaneni","Perry M Elliott","Giampaolo Merlini","Marcia Waddington-Cruz","Arnt V Kristen","Martha Grogan","Ronald Witteles","Thibaud Damy","Brian M Drachman","Sanjiv J Shah","Mazen Hanna","Daniel P Judge","Alexandra I Barsdorf","Peter Huber","Terrell A Patterson","Steven Riley","Jennifer Schumacher","Michelle Stewart","Marla B Sultan","Claudio Rapezzi"],"significance":10,"published":"2018-09-13","source_date":"2018-09-13","image":"","kennis":["https://hartvaat.nl/kennis/hartfalen/betablokkers-bij-hartfalen/"],"congress":"","summary_en":"The ATTR-ACT trial demonstrated that tafamidis significantly reduced all-cause mortality and cardiovascular hospitalization in patients with transthyretin amyloid cardiomyopathy. As the first proven pharmacological treatment for cardiac amyloidosis, this study transformed a previously untreatable condition into a manageable disease.","created":"2026-07-03T10:27:28Z","updated":"2026-07-03T13:26:43Z","licence":"Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.","body_markdown":"Landmark NEJM ATTR-ACT-trial die aantoonde dat tafamidis cardiovasculaire mortaliteit en hospitalisatie significant vermindert bij transthyretine amyloïd cardiomyopathie. Eerste bewezen behandeling voor cardiale amyloïdose.","abstract_original":"BACKGROUND: Transthyretin amyloid cardiomyopathy is caused by the deposition of transthyretin amyloid fibrils in the myocardium. The deposition occurs when wild-type or variant transthyretin becomes unstable and misfolds. Tafamidis binds to transthyretin, preventing tetramer dissociation and amyloidogenesis. METHODS: In a multicenter, international, double-blind, placebo-controlled, phase 3 trial, we randomly assigned 441 patients with transthyretin amyloid cardiomyopathy in a 2:1:2 ratio to receive 80 mg of tafamidis, 20 mg of tafamidis, or placebo for 30 months. In the primary analysis, we hierarchically assessed all-cause mortality, followed by frequency of cardiovascular-related hospitalizations according to the Finkelstein-Schoenfeld method. Key secondary end points were the change from baseline to month 30 for the 6-minute walk test and the score on the Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS), in which higher scores indicate better health status. RESULTS: In the primary analysis, all-cause mortality and rates of cardiovascular-related hospitalizations were lower among the 264 patients who received tafamidis than among the 177 patients who received placebo (P<0.001). Tafamidis was associated with lower all-cause mortality than placebo (78 of 264 [29.5%] vs. 76 of 177 [42.9%]; hazard ratio, 0.70; 95% confidence interval [CI], 0.51 to 0.96) and a lower rate of cardiovascular-related hospitalizations, with a relative risk ratio of 0.68 (0.48 per year vs. 0.70 per year; 95% CI, 0.56 to 0.81). At month 30, tafamidis was also associated with a lower rate of decline in distance for the 6-minute walk test (P<0.001) and a lower rate of decline in KCCQ-OS score (P<0.001). The incidence and types of adverse events were similar in the two groups. CONCLUSIONS: In patients with transthyretin amyloid cardiomyopathy, tafamidis was associated with reductions in all-cause mortality and cardiovascular-related hospitalizations and reduced the decline in functional capacity and quality of life as compared with placebo. (Funded by Pfizer; ATTR-ACT ClinicalTrials.gov number, NCT01994889 .)."}