Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study

Orphanet Journal of Rare Diseases

Research

Chia-Tung Shun¹  Mao-Yuan Su²  Hsueh-Wen Hsueh³  Sung-Tsang Hsieh³  Chi-Chao Chao³  Ming-Jen Lee³  Mei-Fang Cheng⁴  Chia-Hung Chou⁵  Yen-Hung Lin⁶  An-Li Yu⁶  Yuan-Kun (Aden) Wu⁶  Jimmy Jyh-Ming Juang⁶  Ping-Huei Tseng⁷  Cheng-Hsuan Tsai⁸ 

[1] Department of Forensic Medicine and Pathology, National Taiwan University Hospital, Taipei, Taiwan;Department of Pathology, Good Liver Clinic, Taipei, Taiwan;Department of Medical Imaging, National Taiwan University Hospital, Taipei, Taiwan;Department of Medical Imaging and Radiological Technology, Yuanpei University of Medical Technology, Hsinchu, Taiwan;Department of Neurology, National Taiwan University Hospital, Taipei, Taiwan;Department of Nuclear Medicine, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan;Institute of Environmental and Occupational Health Sciences, National Taiwan University, Taipei, Taiwan;Department of Obstetrics and Gynecology, Department of Internal Medicine, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan;Division of Cardiology, National Taiwan University Hospital, Taipei, Taiwan;5Cardiovascular Center, National Taiwan University Hospital, Taipei, Taiwan;Division of Gastroenterology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan;National Taiwan University College of Medicine, Graduate Institute of Clinical Medicine, Taipei, Taiwan;Department of Internal Medicine, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan;

关键词: Cardiac magnetic resonance;    Extracellular volume;    Hereditary transthyretin amyloidosis;    Tafamidis;   

DOI  :  10.1186/s13023-023-02824-0

 received in 2023-02-01, accepted in 2023-07-10,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundTransthyretin cardiac cardiomyopathy (ATTR-CM) is a rare but life-threatening disease. Tafamidis is an effective treatment for patients with ATTR-CM, however its long-term effects on cardiac remodeling and cardiac amyloid deposition are unknown. This study aimed to used cardiac magnetic resonance (CMR) to investigate the effects of tafamidis on patients with hereditary A97S ATTR-CM.MethodsWe retrospectively analyzed a prospective cohort of ATTR-CM patients, including 14 with hereditary A97S ATTR-CM and 17 healthy controls with baseline CMR data. All ATTR-CM patients received tafamidis treatment and received CMR with extracellular volume (ECV) at baseline and after 1 year of follow-up.ResultsBaseline N-terminal pro-B-type natriuretic peptide, left ventricular (LV) mass, LV ejection fraction, global radial, circumferential and longitudinal strain, T1 mapping and ECV were significantly worse in the patients with ATTR-CM compared with the healthy controls. After 1 year of tafamidis treatment, ECV decreased from 51.5 ± 8.9% to 49.0 ± 9.4% (P = 0.041), however there were no significant changes in LV mass, LV ejection fraction, global radial strain, global circumferential strain, global longitudinal strain and T1 mapping.ConclusionsAfter a one-year treatment period, tafamidis exhibited subtle but statistically significant reductions in ECV, potentially indicating a decrease in amyloid deposition among patients diagnosed with hereditary A97S ATTR-CM.

【 授权许可】

CC BY   
© Institut National de la Santé et de la Recherche Médicale (INSERM) 2023

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