**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Giulio Sinigiani, Department of Cardio-Thoraco-Vascular Sciences and Public Health, University of Padua, Padua, Italy

**Main Body of the Abstract**
Background: Tafamidis is currently the only approved disease-modifying therapy for wild-type transthyretin cardiomyopathy (wtATTR-CM). Characteristics of patients who experience disease progression (DP) despite tafamidis therapy are unknown.
Methods: This is a multicentre, prospective study performed in 11 referral centres including patients with wtATTR-CM treated with tafamidis for at least 12 months. The primary endpoint was DP, defined as both increase in NT-proBNP (both >700 ng/L and >30% from baseline levels) and any initiation or increase in the dose of loop diuretic over a 12-month period.
Results: Overall, 297 patients (median age 77 years, 92% males) were recruited. During a 12-month median follow-up (Q1-Q3:12-18), 33 patients (11%) showed DP. Compared with those without, patients with DP had, at baseline, thicker median interventricular septum (IVS) (18 vs 17 mm, p=0.040), presented more frequently with a left ventricular restrictive filling pattern (41% vs 24%, p=0.044), higher E/e’ (18 vs 14, p=0.005) and greater systolic pulmonary artery pressure (sPAP) (40 vs 36 mmHg, p=0.039), and a worse tricuspid annulus planar excursion (TAPSE) to sPAP ratio (0.41 vs 0.47 mm/mmHg, p=0.049) (Figure 1). No significant differences in age, NT-proBNP and creatinine levels between the two groups were observed. Patients with DP showed a higher incidence of heart failure hospitalization (9.1% vs 2%/100pts/year, p=0.003) and of all-cause death (6.5% vs 1.0%/100pts/year, p=0.004) over a 22-month period.
Conclusions: DP is observed in 10% of wtATTR-CM patients treated with tafamidis and is associated with worse outcome. Echocardiographic features of a more advanced disease phenotype are associated with DP.



**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Giulio Sinigiani, Department of Cardio-Thoraco-Vascular Sciences and Public Health, University of Padua, Padua, Italy

**Main Body of the Abstract**
**Background**: Tafamidis is currently the only approved disease-modifying therapy for wild-type transthyretin cardiomyopathy (wtATTR-CM). Characteristics of patients who experience disease progression (DP) despite tafamidis therapy are unknown.
**Methods:** This is a multicentre, prospective study performed in 11 referral centres including patients with wtATTR-CM treated with tafamidis for at least 12 months. The primary endpoint was DP, defined as both increase in NT-proBNP (both >700 ng/L and >30% from baseline levels) and any initiation or increase in the dose of loop diuretic over a 12-month period.
**Results:** Overall, 297 patients (median age 77 years, 92% males) were recruited. During a 12-month median follow-up (Q1-Q3:12-18), 33 patients (11%) showed DP. Compared with those without, patients with DP had, at baseline, thicker median interventricular septum (IVS) (18 vs 17 mm, p=0.040), presented more frequently with a left ventricular restrictive filling pattern (41% vs 24%, p=0.044), higher E/e’ (18 vs 14, p=0.005) and greater systolic pulmonary artery pressure (sPAP) (40 vs 36 mmHg, p=0.039), and a worse tricuspid annulus planar excursion (TAPSE) to sPAP ratio (0.41 vs 0.47 mm/mmHg, p=0.049) **(Figure 1)**. No significant differences in age, NT-proBNP and creatinine levels between the two groups were observed. Patients with DP showed a higher incidence of heart failure hospitalization (9.1% vs 2%/100pts/year, p=0.003) and of all-cause death (6.5% vs 1.0%/100pts/year, p=0.004) over a 22-month period.
**Conclusions:** DP is observed in 10% of wtATTR-CM patients treated with tafamidis and is associated with worse outcome. Echocardiographic features of a more advanced disease phenotype are associated with DP.



Disease progression in patients with wild-type transthyretin cardiomyopathy on treatment with tafamidis: prevalence and clinical correlates. PC133 (#374)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
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