Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy

• الحاوية / القاعدة: Cardiology and Therapy
• المؤلفون الرئيسيون: Zhuang Tian, Daoquan Peng, Wei Ma, Jiangtao Yan, Jian’an Wang, Yida Tang, Wei Jin, Ying Liu, Caiping Jia, Yingxu Gao, Yankun Gong, Xiaohong Sun, Naihan Chen, Shuiqing Zhu, Shuyang Zhang
• التنسيق: مقال
• اللغة: الإنجليزية
• منشور في: Adis, Springer Healthcare 2025-05-01
• الموضوعات: Heart failure; Amyloidosis; Quality of life; Health status; Functional status; Adverse events
• الوصول للمادة أونلاين: https://doi.org/10.1007/s40119-025-00408-6
• تدمد: 2193-8261; 2193-6544

Abstract Introduction Tafamidis is approved in many countries for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Approval is largely based on findings from an international phase 3 trial. This post-approval commitment study aimed to evaluate the safety and efficacy of tafamidis in patients with ATTR-CM in China. Methods A multicenter, single-arm study in Chinese patients with symptomatic ATTR-CM in China. All patients received once-daily, open-label tafamidis free acid 61 mg for 12 months. Safety reporting was ongoing with efficacy assessments at months 6 and 12, including 6-min walk test distance, New York Heart Association (NYHA) functional classification, National Amyloidosis Centre staging, N-terminal pro-B-type natriuretic peptide and troponin I concentrations, Kansas City Cardiomyopathy Questionnaire Overall Summary score, 5-level EQ-5D index score, EQ visual analog scale, and 12-item Short Form Survey. Results Patients (n = 53) were aged 60 (standard deviation [SD]: 12) years, 89% were male, and 94% had variant ATTR-CM (21% had A97S [p.A117S]). At baseline, most (81%) patients had NYHA class II symptoms (6% class I; 13% class III) and National Amyloidosis Centre stage I disease (74%; 21% stage II; 6% stage III). Median treatment exposure was 345 (range, 24‒418) days. Overall, 85% of patients reported treatment-emergent adverse events (TEAEs). The nature and incidence of TEAEs were consistent with the known safety profile of tafamidis. There were no serious or severe treatment-related TEAEs. At 6 and 12 months, there were minimal changes from baseline in all efficacy outcomes with tafamidis, and a high proportion of patients (≥ 44%) showed clinically relevant stability or improvement in each measure. Conclusions The safety of tafamidis in Chinese patients with ATTR-CM was consistent with that previously determined. Tafamidis treatment was associated with a stable disease profile over 12 months in a population of patients where most had variant ATTR-CM and mild heart failure symptoms. Trial Registration NCT04814186.