Transthyretin cardiac amyloidosis (ATTR-CM) is caused by the accumulation of misfolded transthyretin (TTR) protein in the myocardium. Diflunisal, an agent that stabilizes TTR, has been used as an off-label therapeutic for ATTR-CM. Given limited data surrounding the use of diflunisal, a systematic review of the literature is warranted. We searched the PubMed, MEDLINE, and Embase databases for studies that reported on the use of diflunisal therapy for patients with ATTR-CM. We included English language studies which assessed the effect of diflunisal in adult patients with ATTR-CM who received diflunisal as primary treatment and reported clinical outcomes with emphasis on studies that noted the safety and efficacy of diflunisal in cardiac manifestations of ATTR amyloidosis. We excluded studies which did not use diflunisal therapy or used diflunisal therapy for non-cardiac manifestations of TTR amyloidosis. We also excluded case reports, abstracts, oral presentations, and studies with fewer than 10 subjects. Our search yielded 316 records, and we included 6 studies reporting on 400 patients. Non-comparative single-arm small non-randomized trials for diflunisal comprised 4 of the included studies. The 2 studies that compared diflunisal versus no treatment found improvements in TTR concentration, left atrial volume index, cardiac troponin I, and global longitudinal strain. Overall, diflunisal use was associated with decreased mortality and number of orthotopic heart transplant in ATTR-CM patients. Although a smaller number of patients had to stop treatment due to gastrointestinal side effects and transient renal dysfunction, there were no severe reactions reported in the studies included in our review. This systematic review supports the use of diflunisal for ATTR-CM. Additional long-term analyses and randomized clinical trials are needed to confirm these results.

转甲状腺素蛋白心脏淀粉样变性 (ATTR-CM) 是由心肌中错误折叠的转甲状腺素蛋白 (TTR) 蛋白的积累引起的。Diflunisal 是一种稳定 TTR 的药物，已被用作 ATTR-CM 的标签外治疗剂。鉴于围绕使用二氟尼柳的数据有限，有必要对文献进行系统回顾。我们在 PubMed、MEDLINE 和 Embase 数据库中搜索了报告使用双氟尼柳治疗 ATTR-CM 患者的研究。我们纳入了英语语言研究，这些研究评估了二氟尼沙对接受二氟尼沙作为主要治疗的成年 ATTR-CM 患者的影响，并报告了临床结果，重点是指出二氟尼沙在 ATTR 淀粉样变性心脏表现中的安全性和有效性的研究。我们排除了未使用二氟尼柳疗法或使用二氟尼柳疗法治疗 TTR 淀粉样变性的非心脏表现的研究。我们还排除了病例报告、摘要、口头报告和少于 10 名受试者的研究。我们的搜索产生了 316 条记录，我们纳入了 6 项研究，报告了 400 名患者。双氟尼柳的非比较性单臂小型非随机试验包括 4 项纳入研究。比较二氟尼柳与不治疗的 2 项研究发现 TTR 浓度、左心房容积指数、心肌肌钙蛋白 I 和整体纵向应变有所改善。总体而言，二氟尼柳的使用与 ATTR-CM 患者的死亡率和原位心脏移植数量降低有关。尽管少数患者因胃肠道副作用和短暂的肾功能不全而不得不停止治疗，但在我们审查的研究中没有报告严重的反应。该系统评价支持将二氟尼柳用于 ATTR-CM。需要额外的长期分析和随机临床试验来证实这些结果。