González-López Esther, López-Sainz Ángela, Garcia-Pavia Pablo
Unidad de Insuficiencia Cardiaca y Cardiopatías Familiares, Servicio de Cardiología, Hospital Universitario Puerta de Hierro, Majadahonda, Madrid, Spain; Centro de Investigación Biomédica en Red de Enfermedades Cardiovasculares (CIBERCV), Madrid, Spain.
Unidad de Insuficiencia Cardiaca y Cardiopatías Familiares, Servicio de Cardiología, Hospital Universitario Puerta de Hierro, Majadahonda, Madrid, Spain.
Rev Esp Cardiol (Engl Ed). 2017 Nov;70(11):991-1004. doi: 10.1016/j.rec.2017.05.036. Epub 2017 Sep 1.
Cardiac amyloidosis is an infiltrative disorder caused by extracellular protein deposition. Transthyretin is a proamyloidotic protein that produces one of the most frequent forms of cardiac amyloidosis, either through mutations or a wild-type form (previously known as senile amyloidosis). Until very recently, diagnosis of transthyretin amyloidosis (ATTR) was very uncommon and histological confirmation was mandatory, making diagnosis of ATTR a real challenge in daily clinical practice. Moreover, the specific therapeutic options to alter the clinical course of the disease were very limited. However, advances in cardiac imaging and diagnostic strategies have improved recognition of ATTR. In addition, several compounds able to modify the natural history of the disease are in the final phases of research, with promising results. Given that effective therapies are on the horizon, cardiologists should be well-versed in this disease and be familiar with its diagnosis and treatment. This review describes the broad clinical spectrum of ATTR in detail, as well as recent advances in the diagnosis and treatment of this condition.
心脏淀粉样变性是一种由细胞外蛋白质沉积引起的浸润性疾病。转甲状腺素蛋白是一种促淀粉样变性蛋白,它通过突变或野生型形式(以前称为老年性淀粉样变性)产生最常见的心脏淀粉样变性形式之一。直到最近,转甲状腺素蛋白淀粉样变性(ATTR)的诊断还非常少见,组织学确诊是必需的,这使得ATTR的诊断在日常临床实践中成为一项真正的挑战。此外,改变疾病临床进程的特定治疗选择非常有限。然而,心脏成像和诊断策略的进展提高了对ATTR的认识。此外,几种能够改变疾病自然史的化合物正处于研究的最后阶段,取得了令人鼓舞的结果。鉴于有效的治疗方法即将出现,心脏病专家应该精通这种疾病,并熟悉其诊断和治疗。这篇综述详细描述了ATTR广泛的临床谱,以及该疾病诊断和治疗方面的最新进展。