Abstract 21: Characteristics Of Patients With Transthyretin Amyloid Cardiomyopathy Receiving Tafamidis Therapy: Experience At An Advanced Heart Failure Program

Abstract

Background: While the safety and efficacy of tafamidis are described in clinical trials, data on its real-world application are lacking. This study summarized the characteristics of patients initiating first-time treatment with tafamidis at the Cardiac Amyloidosis Clinic at Georgia Heart Institute. Methods: A retrospective analysis of the clinical characteristics and treatment progress of patients diagnosed with transthyretin amyloid (ATTR) cardiomyopathy receiving tafamidis therapy from May 6, 2019, to Oct 1, 2021. Results: In total, 18 patients were identified who had received tafamidis. Two patients were excluded from the analysis as they did not receive care from our clinic. Of the remaining 16 patients, 2 African American and 1 Caucasian patients with a mean age of 59.5 years old, carried V122I mutation. The other 13 patients, Caucasian with a mean age of 81.4 years old, possessed wild-type ATTR amyloidosis. No patients with V30M mutation were identified. Of the patients included, 14 male and 2 female, the 2 female patients had wild-type ATTR amyloidosis. At the time of tafamidis initiation, 10 patients were at the UK National Amyloidosis Centre (NAC) stage 1 amyloid cardiomyopathy, 5 patients at NAC stage 2, and 1 patient at NAC stage 3. The median (IQR) levels of NT-proBNP were 2240 (1098 - 4148) pg/mL at baseline, and 1446 (800 - 2795) pg/mL at 12 months after initiation of therapy. The mean eGFR of patients with wild-type ATTR and V122I mutation were 58 and 72 mL/min/1.73 m2, respectively. Four patients received additional therapy with 1) doxycycline, 2) doxycycline and tauroursodeoxycholic acid, 3) diflunisal, 4) patisiran. There were 13.33% patients on beta-blockers, 53.33% received ACEI/ARBs, and 86.67% took aldosterone antagonists. No adverse events were reported nor therapy discontinuation. Conclusion: Our findings are consistent with published data with wild-type ATTR more frequently seen in males and V122I mutation predominantly seen in individuals of African descent. Overall, in a real-world setting, tafamidis was associated with a decline in NT-proBNP and well-tolerated in our patients.