Introduction: Transthyretin cardiac amyloidosis (ATTR) is an infiltrative cardiomyopathy that frequently presents with heart failure, aortic stenosis, and conduction abnormalities. Tafamidis, a transthyretin stabiliser, has shown efficacy in preventing amyloid formation and is the first specific treatment approved in Spain for this condition. The ATTR-ACT study, published in 2018, demonstrated significant clinical and prognostic benefits with this drug.
Objectives: The aim of this study was to describe the clinical and demographic profile of patients with ATTR treated with tafamidis in our area, to evaluate their clinical and analytic evolution, and to compare these results with those reported in the ATTR-ACT study.
Methods: We generated a case series in which we collected demographic characteristics of the patients, as well as certain laboratory values before and after tafamidis, 6-minute walk test (6MWT) results, and usual administered treatment.
Results: Results are summarised in Table 1.
Discussion: The results showed that the demographic characteristics of the patients in our series were comparable to those in the ATTR- ACT study, with a median age of 78 years versus 75 years in the original study, and a similar male predominance (93% vs. 91%). Adverse effects were more frequent in our series, which may be related to the smaller sample size. However, as in the ATTR-ACT study, gastrointestinal disturbances were the most commonly reported side effects. Regarding baseline functional class (FC), the results were also similar, with a greater representation of FC III in ATTR-ACT study.
Although clinical progression was not analysed due to the short follow-up period in our series, a slight decrease in NT-proBNP level was observed over time. Most prescriptions were made by internal medicine specialists, despite of the fact that initial diagnoses were commonly established during cardiology hospitalisations, highlighting the importance of a multidisciplinary approach in managing these patients.
Finally, comorbidity and dependency scales should be considered before prescribing tafamidis, as it is an expensive treatment and its benefit is more pronounced in the long term.
