{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["14(3)"],"submitter":["Tian Z"],"funding":["Pfizer"],"pubmed_abstract":["<h4>Introduction</h4>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.<h4>Methods</h4>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.<h4>Results</h4>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.<h4>Conclusions</h4>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.<h4>Trial registration</h4>NCT04814186."],"journal":["Cardiology and therapy"],"pagination":["439-452"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12378879"],"repository":["biostudies-literature"],"pubmed_title":["Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy."],"pmcid":["PMC12378879"],"pubmed_authors":["Chen N","Jin W","Yan J","Jia C","Sun X","Zhang S","Wang J","Gong Y","Ma W","Tian Z","Liu Y","Peng D","Gao Y","Tang Y","Zhu S"],"additional_accession":[]},"is_claimable":false,"name":"Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy.","description":"<h4>Introduction</h4>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.<h4>Methods</h4>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.<h4>Results</h4>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.<h4>Conclusions</h4>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.<h4>Trial registration</h4>NCT04814186.","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Sep","modification":"2026-05-10T04:24:32.02Z","creation":"2026-04-08T01:28:35.095Z"},"accession":"S-EPMC12378879","cross_references":{"pubmed":["40410537"],"doi":["10.1007/s40119-025-00408-6"]}}