Tafamidis treatment may decrease the levels of N-terminal pro-brain natriuretic peptide (NT-proBNP) in hereditary transthyretin amyloidosis (hATTR) with cardiac involvement caused by the A97S mutation, and it appears particularly beneficial for patients with a severe form of the disease, according to a study published in Acta Cardiologica Sinica.

The researchers retrospectively analyzed the records of 20 patients with A97S hATTR with cardiac involvement treated with 61 mg of tafamidis per day at the National Taiwan University Hospital. All patients underwent functional status, biochemistry, and echocardiography testing at baseline and after 6 months of receiving tafamidis.

The study evaluated NT-proBNP levels at baseline and after 6 months of tafamidis treatment as the primary outcome measure.

According to the results, baseline NT-proBNP levels of 2.9±0.6 improved to 2.7±0.5 after 6 months of tafamidis treatment. Subgroup analysis showed higher levels of left ventricle posterior wall thickness and left atrial diameter among patients with improved NT-proBNP levels.

Read more about hATTR treatment

“This finding suggests the short-term benefit of tafamidis in the patients with worse cardiac involvement, possibly because these patients had higher baseline NT-proBNP,” Tsai and colleagues explained.

Baseline left ventricular mass index was 200.9±63.9 g/m2 and baseline LV ejection fraction was 58.9±13.5%. Serum transthyretin levels also increased significantly upon tafamidis treatment.

“A longer duration of tafamidis treatment may be needed to demonstrate the therapeutic effects in patients with lower baseline NT-proBNP and less cardiac involvement of amyloidosis. Further larger studies with longer follow-up are needed to confirm this finding,” the authors emphasized.

A97S (p.Ala117Ser), the most common transthyretin genetic mutation in Taiwan, remains poorly investigated. Patients often experience refractory heart failure, as traditional heart failure management yields limited benefits in cardiac A97S hATTR and may even be harmful due to hypotension and concomitant autonomic dysfunction.


Tsai CH, Yu AL, Wu YK, et al. Efficacy of tafamidis in patients with Ala97Ser hereditary transthyretin cardiac amyloidosis: a six-month follow-up study. Acta Cardiol Sin. Published online July 2023. doi:10.6515/ACS.202307_39(4).20221116A