Sukaina Mahnoor, Rehman Shafi, Waheed Marium, Shehryar Muhammad, Rasool Raffat, Ahmed Nimra, Ochani Sidhant, Hasibuzzaman Md Al
Department of Medicine, Karachi Medical and Dental College, Karachi.
Department of Medicine, Khyber Medical College, Peshawar.
Ann Med Surg (Lond). 2023 Nov 7;86(1):433-438. doi: 10.1097/MS9.0000000000001482. eCollection 2024 Jan.
In May 2019, the U.S. Food and Drug Administration approved tafamidis as the first conservative management of transthyretin amyloid cardiomyopathy (ATTR-CM). Our aim in conducting this systematic review and meta-analysis was to assess the efficacy of tafamidis on patients with ATTR-CM. For that purpose, we thoroughly searched PubMed, ScienceDirect, and Clinical trails.gov by using the appropriate search strategy and following predefined inclusion and exclusion criteria, which retrieved 235 articles initially. Of which two randomized controlled trials (RCTs) and one observational study matched our inclusion criteria. A total of 876 patients are included in this analysis. Based on results, tafamidis significantly reduced cardiovascular (CV) mortality in the ATTR-ACT trial and Ochi . (OR 0.58; 95% CI: [0.41-0.83], =0.003, =87%). A subgroup analysis was conducted for CV mortality due to heart failure (OR 0.89; 95% CI: [0.63-1.25], =0.50, =93%). The results exhibit that tafamidis reduced all causes of mortality (OR 0.45; 95% CI: [0.32-0.64], ≤0.00001, =22%). Furthermore, mortality remained statistically insignificant in patients with heart transplants (OR 1.18; 95% CI: [0.52-2.70], =0.70, =0%) and patients with cardiac mechanical assist devices (OR 4.15; 95% CI: [0.48-35.66], =0.20, =0%). This meta-analysis suggests that tafamidis is a safe and efficient drug to use in patients with ATTR-CM and can possess the potential to be a milestone in enhancing the conservative management of the patients.
2019年5月，美国食品药品监督管理局批准了氯苯唑酸，作为转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）的首个保守治疗药物。我们开展这项系统评价和荟萃分析的目的是评估氯苯唑酸对ATTR-CM患者的疗效。为此，我们采用适当的检索策略，按照预先设定的纳入和排除标准，全面检索了PubMed、ScienceDirect和Clinical trials.gov，初步检索到235篇文章。其中两项随机对照试验（RCT）和一项观察性研究符合我们的纳入标准。本分析共纳入876例患者。基于研究结果，在ATTR-ACT试验和Ochi研究中，氯苯唑酸显著降低了心血管（CV）死亡率（OR 0.58；95%CI：[0.41 - 0.83]，P = 0.003，I² = 87%）。对因心力衰竭导致的CV死亡率进行了亚组分析（OR 0.89；95%CI：[0.63 - 1.25]，P = 0.50，I² = 93%）。结果显示，氯苯唑酸降低了全因死亡率（OR 0.45；95%CI：[0.32 - 0.64]，P≤0.00001，I² = 22%）。此外，在心脏移植患者（OR 1.18；95%CI：[0.52 - 2.70]，P = 0.70，I² = 0%）和使用心脏机械辅助装置的患者（OR 4.15；95%CI：[0.48 - 35.66]，P = 0.20，I² = 0%）中，死亡率在统计学上无显著差异。这项荟萃分析表明，氯苯唑酸是一种用于ATTR-CM患者的安全有效的药物，并且有可能成为加强患者保守治疗的一个里程碑。
