Tafamidis treatment in patients with transthyretin amyloid cardiomyopathy: a systematic review and meta-analysis

Jie Wang; Hongyu Chen; Zihuan Tang; Jinquan Zhang; Yuanwei Xu; Ke Wan; Kifah Hussain; Georgios V Gkoutos; Yuchi Han; Yucheng Chen

doi:10.1016/j.eclinm.2023.102172

Tafamidis treatment in patients with transthyretin amyloid cardiomyopathy: a systematic review and meta-analysis

EClinicalMedicine. 2023 Aug 24:63:102172. doi: 10.1016/j.eclinm.2023.102172. eCollection 2023 Sep.

Authors

Jie Wang^{1

2}, Hongyu Chen³, Zihuan Tang³, Jinquan Zhang⁴, Yuanwei Xu¹, Ke Wan⁵, Kifah Hussain⁶, Georgios V Gkoutos^{2

7

8

9}, Yuchi Han¹⁰, Yucheng Chen^{1

11}

Affiliations

¹ Department of Cardiology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
² College of Medical and Dental Sciences, Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK.
³ West China School of Public Health, Sichuan University, Chengdu, Sichuan, China.
⁴ Division of Informatics, Imaging, and Data Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
⁵ Department of Geriatrics, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
⁶ Department of Cardiology, NorthShore University Health Systems, Evanston, Illinois, USA.
⁷ Institute of Translational Medicine, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
⁸ Health Data Research UK (HDR), Midlands Site, UK.
⁹ Centre for Health Data Science, University of Birmingham, Birmingham, UK.
¹⁰ Cardiovascular Division, Wexner Medical Centre, The Ohio State University, USA.
¹¹ Centre of Rare Diseases, West China Hospital, Sichuan University, Chengdu, Sichuan, China.

Abstract

Background: Previous studies have reported that tafamidis treatment was associated with better outcomes in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) compared with those without tafamidis treatment. Therefore, we aimed to systematically assess the association of tafamidis treatment with outcomes in patients with ATTR-CM.

Methods: The protocol for this systematic review and meta-analysis was registered in the PROSPERO (CRD42022381985). Pubmed, Ovid Embase, Scopus, Cochrane Library, and Web of Science were interrogated to identify studies that evaluated the impact of tafamidis on prognosis in ATTR-CM, from January 1, 2000 to June 1, 2023. A random-effects model was used to determine the pooled risk ratio (RR) for the adverse endpoints. In addition, the main outcomes included all-cause death or heart transplantation, the composite endpoints included all-cause death, heart transplantation, cardiac-assist device implantation, heart failure exacerbations, and hospitalization.

Findings: Fifteen studies comprising 2765 patients (mean age 75.9 ± 9.3 years; 83.7% male) with a mean follow-up duration of 18.7 ± 17.1 months were included in the meta-analysis. There was a decrease in left ventricular ejection fraction (LVEF) (standard mean differences (SMD: -0.17; 95% confidence interval (CI), -0.31 to -0.03; P = 0.02) but were no significant differences in intraventricular septum (IVS) thickness or global longitudinal strain (GLS) after tafamidis treatment. However, subgroup analysis showed no significant deterioration in LVEF in the patients with wild-type ATTR after tafamidis treatment (SMD: -0.11; 95% CI, -0.34 to 0.12, P = 0.34). In addition, the group with tafamidis treatment had a decreased risk for all-cause death or heart transplantation compared to patients without treatment (the pooled RR, 0.44; 95% CI, 0.31-0.65; P < 0.01). Subgroup analysis showed that there was no significant difference of tafamidis on the outcomes in patients with wild-type or hereditary ATTR (RR, 0.44; 95% CI, 0.27-0.73 versus 0.21, 95% CI, 0.11-0.40, P = 0.08). Furthermore, tafamidis treatment was associated with a lower risk of the composite endpoint (RR, 0.57; 95% CI, 0.42-0.77; P < 0.01).

Interpretation: Our findings suggested that there was no significant deterioration in LVEF in the patients with wild-type ATTR after tafamidis treatment. In addition, tafamidis treatment was associated with a low risk of all-cause death and adverse cardiovascular events.

Funding: This work was supported by grants from the Natural Science Foundation of Sichuan Province [Grant Number: 23NSFSC4589] and the National Natural Science Foundation of China [Grant Number: 82202248].

Keywords: ATTR; Prognosis; Tafamidis; Transthyretin amyloid cardiomyopathy.