18-Month effect of tafamidis on the progression of cardiac amyloidosis evaluated according to a multiparametric expert consensus tool

Abstract

Background: A recently published expert consensus document recommended a multiparametric tool to monitor cardiac disease progression in patients with transthyretin cardiac amyloidosis (ATTR-CA). We aimed to evaluate the effect of the transthyretin stabiliser drug, tafamidis, by applying this integrative tool. Methods: We retrospectively applied a multiparametric tool in a group of ATTR-CA patients who were given tafamidis between the years 2019–2021 and were followed in a dedicated clinic. We used three pre-specified follow-up timepoints: at 6, 12 and 18 months. Results: We included 16 ATTR-CA patients (wild-type (n = 14) and mutant (n = 2)). The median age at the initiation of tafamidis was 76 (IQR 70, 84) years and 75% of study patients were classified as NYHA functional class 2 or 3. All patients had elevated levels of high-sensitive troponin T (median 92 (IQR 63, 115) ng/L) and NT-proBNP (median 3784 (2290, 8773) pg/mL). At the end of 18-month follow-up, two patients have suffered from high-grade atrioventricular block and required permanent pacing, and one patient had heart-failure-related admission. Twenty-five percent and 50% of patients were classified as NYHA Class 1 at the initiation of tafamidis and at 18-months treatment, respectively. No patient was defined with disease progression at 6- or 12-month follow up; however, one patient (14%) was defined with a deteriorated disease status at 18-month follow-up. Conclusions: Based on a multiparametric tool, the use of tafamidis promoted disease stabilisation in the majority of patients at 18-month follow-up. Further study should focus on monitoring disease improvement in patients with ATTR-CA.

Keywords:

• Tafamidis
• transthyretin cardiac amyloidosis
• prognosis
• amyloidosis

Disclosure statement

RK – declare no conflict of interests.

OI – has served on advisory boards for Pfizer and has received speaking fees from Pfizer.

All authors have participated in the work and have reviewed and agree with the content of the article. None of the article contents are under consideration for publication in any other journal or have been published in any journal. No portion of the text has been copied from other material in the literature.

Additional information

Funding

None.