ATTR Amyloidosis: Current and Emerging Management Strategies: JACC: CardioOncology State-of-the-Art Review.
ATTR Amyloidosis: Current and Emerging Management Strategies: JACC: CardioOncology State-of-the-Art Review.
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DOI：
10.1016/j.jaccao.2021.06.006
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发表时间：
2021-10
期刊：
影响因子：
--
通讯作者：
Cheng RK
中科院分区：
文献类型：
--
作者：
Griffin JM;Rosenthal JL;Grodin JL;Maurer MS;Grogan M;Cheng RK
甲状腺素运载蛋白心脏淀粉样变性（ATTR-CA）的诊断越来越多，由于非侵入性成像的出现和提高认识。致病等位基因的临床表现不完全，因此存在大量无症状甲状腺素运载蛋白变异携带者。亚临床ATTR-CA的筛查策略、监测和治疗需要进一步研究。也许最重要的转化胜利是从ATTR-CA病理生理学的生物学理解中出现的有效疗法的发展。这些包括最近证实的甲状腺素运载蛋白稳定化和沉默甲状腺素运载蛋白产生的策略。关于ATTR-CA中神经激素阻滞的数据有限，药物治疗的主要重点是明智的液体管理。房颤是常见的，由于血栓形成的倾向，需要抗凝治疗。虽然传导疾病和室性心律失常经常发生，但关于最佳管理知之甚少。最后，主动脉瓣狭窄和ATTR-CA经常共存，经导管瓣膜置换术是首选的治疗方法。ATTR-CA越来越多地认识到，由于提高认识和非侵入性诊断途径的出现。致病等位基因的临床检出率不完全，用于筛查或管理无症状携带者的数据有限。对ATTR-CA病理学的生物学理解已经导致TTR稳定剂和沉默剂的有效靶向疗法。潜在的未来治疗包括CRISPR，淀粉样蛋白提取/降解和抑制淀粉样蛋白播种。
Transthyretin cardiac amyloidosis (ATTR-CA) is increasingly diagnosed owing to the emergence of noninvasive imaging and improved awareness. Clinical penetrance of pathogenic alleles is not complete and therefore there is a large cohort of asymptomatic transthyretin variant carriers. Screening strategies, monitoring, and treatment of subclinical ATTR-CA requires further study. Perhaps the most important translational triumph has been the development of effective therapies that have emerged from a biological understanding of ATTR-CA pathophysiology. These include recently proven strategies of transthyretin protein stabilization and silencing of transthyretin production. Data on neurohormonal blockade in ATTR-CA are limited, with the primary focus of medical therapy on judicious fluid management. Atrial fibrillation is common and requires anticoagulation owing to the propensity for thrombus formation. Although conduction disease and ventricular arrhythmias frequently occur, little is known regarding optimal management. Finally, aortic stenosis and ATTR-CA frequently coexist, and transcatheter valve replacement is the preferred treatment approach. ATTR-CA is increasingly recognized owing to improved awareness and emergence of noninvasive diagnostic pathways. Clinical penetrance of pathogenic alleles is incomplete, with limited data for screening or management of asymptomatic carriers. Biological understanding of ATTR-CA pathology has led to effective targeted therapies of TTR stabilizers and silencers. Potential future treatments include CRISPR, amyloid extraction/degradation, and inhibition of amyloid seeding.
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影响因子：
120.7
作者：
Berk, John L.;Suhr, Ole B.;Obici, Laura;Sekijima, Yoshiki;Zeldenrust, Steven R.;Yamashita, Taro;Heneghan, Michael A.;Gorevic, Peter D.;Litchy, William J.;Wiesman, Janice F.;Nordh, Erik;Corato, Manuel;Lozza, Alessandro;Cortese, Andrea;Robinson-Papp, Jessica;Colton, Theodore;Rybin, Denis V.;Bisbee, Alice B.;Ando, Yukio;Ikeda, Shu-ichi;Seldin, David C.;Merlini, Giampaolo;Skinner, Martha;Kelly, Jeffery W.;Dyck, Peter J.
通讯作者：
Dyck, Peter J.
利尿剂剂量和NYHA功能类别是经胸蛋白心脏淀粉样变性患者死亡率的独立预测指标。
DOI：
10.1016/j.jaccao.2020.06.007
发表时间：
2020-09
期刊：
JACC. CardioOncology
影响因子：
--
作者：
Cheng RK;Levy WC;Vasbinder A;Teruya S;De Los Santos J;Leedy D;Maurer MS
通讯作者：
Maurer MS
DOI：
10.2147/dddt.s96893
发表时间：
2015
期刊：
Drug design, development and therapy
影响因子：
--
作者：
aus dem Siepen F;Bauer R;Aurich M;Buss SJ;Steen H;Altland K;Katus HA;Kristen AV
通讯作者：
Kristen AV
影响因子：
5.1
作者：
Brannagan, T. H.;Wang, A. K.;Gertz, M.
通讯作者：
Gertz, M.
影响因子：
120.7
作者：
Damrauer, Scott M.;Chaudhary, Kumardeep;Do, Ron
通讯作者：
Do, Ron