patisiran (onpattro) and poly neuropathy associated with transthyretin amyloidosis

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patisiran (onpattro) and poly neuropathy associated with transthyretin amyloidosis

机译：Patisiran（Onpattro）和与Transthyretin淀粉样蛋白症相关的聚神经病症

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In patients with hereditary transthyretin amyloidosis manifesting as polyneuropathy, and still able to walk with or without assistance, a double-blind placebo-controlled trial showed that treatment with patisiran for 18 months slowed the progression of polyneuropathy, particularly with respect to walking. It is not known whether or not patisiran is effective against other effects of the disease, in particular cardiac involvement, or whether it extends lifespan. Patisiran mainly carries a risk of mild to moderate infusion reactions, extravasation (which can be serious), and possibly liver disorders. Ocular disorders should be monitored. Despite the many uncertainties in the long-term, patisiran is an option worth offering to these patients.

机译：对于表现为多发性神经病的遗传性转甲状腺素淀粉样变性患者，在有或无辅助的情况下仍能行走，一项双盲安慰剂对照试验表明，帕西兰治疗18个月可减缓多发性神经病的进展，尤其是在行走方面。目前尚不清楚帕西兰是否能有效对抗该疾病的其他影响，尤其是心脏受累，或是否能延长寿命。帕蒂西兰主要有轻度至中度输液反应、外渗（可能很严重）以及可能的肝脏疾病的风险。应监测眼部疾病。尽管长期存在许多不确定性，但帕西兰是值得为这些患者提供的选择。

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《Prescrire international》 |2020年第217期|共1页
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1. Symptomatic Val122del mutated hereditary transthyretin amyloidosis:Need for early diagnosis and prioritization for heart and liver transplantation [J] . Adriano-Valerio Schettini, Joan Fabregat, John J Poterucha, 国际肝胆胰疾病杂志（英文版） . 2021,第004期
2. Wild type transthyretin amyloidosis,a reason not to be forgotten for heart failure of preserved ejection fraction in the elderly [J] . Zhuang TIAN, Chao REN, Li HUO, 老年心脏病学杂志（英文版） . 2020,第012期
3. A Review of Patisiran (ONPATTRO?) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis [J] . Ivan Urits, Daniel Swanson, Michael C. Swett, Neurology and therapy. . 2020,第2期

机译：对Patisiran（OnPattro？）治疗遗传性Transthyretin淀粉样蛋白病
4. Patisiran in hereditary transthyretin-mediated amyloidosis [J] . Luigetti Marco, Servidei Serenella Lancet Neurology . 2021,第1期

机译：Patisiran在遗传性的Transthyretin介导的淀粉样蛋白病
5. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study [J] . Adams David, Polydefkis Michael, Gonzalez-Duarte Alejandra, Lancet Neurology . 2021,第1期

机译：Patisiran对遗传性Transthyretin介导的淀粉样蛋白病的长期安全性和有效性，具有多变病变：12个月的开放标签扩展研究结果
6. COMPARISON OF CARDIAC AMYLOIDOSIS ASSOCIATED WITH WILD TYPE TRANSTHYRETIN AND THE ISOLEUCINE 122 VARIANT [C] . H.J. Lachmann, H.J.B.. Goodman, J.D. Gillmore, International Symposium on Amyloidosis . 2008

机译：与野生型Transthyretin和异吲哚122变体相关的心脏淀粉样蛋白病的比较
7. The Role of Non-Coding Genetic Variants on Transthyretin Gene Transcription in Transthyretin Amyloidosis [D] . Boldbaatar, Batbold. 2022

机译：非编码遗传变异在促甲肾上腺素淀粉样蛋白症中的Transthyretin基因转录中的作用
8. A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis [O] . Ivan Urits, Daniel Swanson, Michael C. Swett, 2020

机译：Patisiran（OnPattro®）对遗传性胸腺炎淀粉样蛋白症的人们治疗多肺病变的综述
9. Correction to: A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis [O] . Ivan Urits, Daniel Swanson, Michael C. Swett, 2021

机译：纠正：对Patisiran（OnPattro®）进行审查，用于治疗遗传性Transthyretin淀粉样蛋白症的人们的多发性病变
10. Triphenyl Phosphite Neuropathy Differs from Organophosphorus-Induced Delayed Neuropathy in Rats [R] . Veronesi, B. , Dvergsten, C. 1987

机译：三苯基亚磷酸酯神经病变不同于大鼠中有机磷诱导的延迟性神经病变

patisiran (onpattro) and poly neuropathy associated with transthyretin amyloidosis

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