Transthyretin cardiac amyloidosis (ATTR-CA) is typically a late-onset disease caused by the deposit of transthyretin amyloid fibrils throughout the heart. When this occurs, various cardiac sequelae can develop, including hypotension, conduction abnormalities, and valvular lesions. The cardiomyopathy caused by ATTR-CA (ATTR-CM) has proven difficult to treat. Until recently, symptomatic management was the only therapeutic option, and many therapies used to treat congestive heart failure were ineffective or even detrimental to patients with ATTR-CM. In addition, treatment was limited to heart and liver transplantation. As a result, prognosis was poor. Recently, a few drug therapies have come to light as potential treatment modalities for ATTR-CM, most notably tafamidis, sold under the brand names Vyndaqel and Vyndamax. After the phase III Transthyretin Amyloidosis Cardiomyopathy trial displayed the drug's efficacy, it was given breakthrough therapy designation and was approved by the Food and Drug Administration on May 6, 2019, for the treatment of ATTR-CA. This novel therapy, as well as various other therapies in the pipeline, such as inotersen and patisiran, provide hope where, until recently, there was little. Unfortunately, the exorbitant cost of these new therapies may present a barrier to long-term treatment for some patients. However, by further improving diagnostic algorithms and incorporating these new treatments into our existing therapeutic modalities, patients with ATTR-CA should be able to live far longer than previously expected. Finally, further research combining these novel treatment modalities must be done, as they may prove to be additive or even synergistic in their treatment of ATTR amyloidosis.

中文翻译：

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运甲状腺素蛋白心脏淀粉样变性和治疗这种不太罕见的心肌病原因的新疗法。

运甲状腺素蛋白心脏淀粉样变性（ATTR-CA）通常是一种迟发型疾病，由运甲状腺素蛋白淀粉样蛋白原纤维沉积在整个心脏引起。当这种情况发生时，可能会出现各种心脏后遗症，包括低血压、传导异常和瓣膜病变。ATTR-CA (ATTR-CM) 引起的心肌病已被证明难以治疗。直到最近，对症治疗还是唯一的治疗选择，许多用于治疗充血性心力衰竭的疗法对 ATTR-CM 患者无效甚至有害。此外，治疗仅限于心脏和肝脏移植。结果，预后很差。最近，一些药物疗法被发现可以作为 ATTR-CM 的潜在治疗方式，其中最著名的是以 Vyndaqel 和 Vyndamax 品牌销售的 tafamidis。在III期转甲状腺素蛋白淀粉样变性心肌病试验显示该药物的疗效后，该药物被授予突破性疗法认定，并于2019年5月6日获得美国食品和药物管理局批准，用于治疗ATTR-CA。这种新颖的疗法以及正在研发的各种其他疗法，例如inotersen和patisiran，为直到最近还几乎没有希望的地方带来了希望。不幸的是，这些新疗法的高昂成本可能会给一些患者的长期治疗带来障碍。然而，通过进一步改进诊断算法并将这些新疗法纳入我们现有的治疗方式中，ATTR-CA 患者的寿命应该比之前预期的要长得多。最后，必须进一步研究结合这些新的治疗方式，因为它们可能被证明在 ATTR 淀粉样变性的治疗中具有相加甚至协同作用。