{"title":"Update on Amyloid Polyneuropathy and Treatment","authors":"","doi":"10.1007/s11940-024-00780-z","DOIUrl":null,"url":null,"abstract":"<h3>Abstract</h3> <span> <h3>Purpose of Review</h3> <p>The purpose of this review is to summarize currently available and developing diagnostic and treatment options for hereditary transthyretin amyloid polyneuropathy. Transthyretin amyloidosis (ATTR) predominantly manifests with cardiomyopathy and/or peripheral neuropathy, but amyloid deposits may be found in other organs or tissues.</p> </span> <span> <h3>Recent Findings</h3> <p>Currently available treatments include transthyretin gene silencers (for hereditary ATTR peripheral neuropathy only) and transthyretin stabilizers (tafamidis for ATTR cardiomyopathy in the USA, and for both hereditary ATTR peripheral neuropathy and ATTR cardiomyopathy in Europe, Japan, Brazil, and some other countries), and liver transplantation. Gene silencers stop the progression of hereditary ATTR peripheral neuropathy in most patients, and transthyretin stabilizers reduce hospitalizations and mortality in patients with ATTR cardiomyopathy. The use of liver transplantation for ATTR has declined with the availability of more effective therapies, and shortage of available allografts. On the horizon are new treatments already in clinical trials including new gene silencers and gene editing agents, new transthyretin stabilizers, and amyloid removal treatments.</p> </span> <span> <h3>Summary</h3> <p>Recently approved treatments for ATTR have changed its natural history, and additional medications may get approved in the near future. Early diagnosis is still essential to improve treatment outcomes. New management strategies may include combinations of gene silencers, transthyretin stabilizers, gene editing, and amyloid removal agents, but the cost may become the limiting factor.</p> </span>","PeriodicalId":10975,"journal":{"name":"Current Treatment Options in Neurology","volume":"3 1","pages":""},"PeriodicalIF":2.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Current Treatment Options in Neurology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s11940-024-00780-z","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 0
Abstract
Purpose of Review
The purpose of this review is to summarize currently available and developing diagnostic and treatment options for hereditary transthyretin amyloid polyneuropathy. Transthyretin amyloidosis (ATTR) predominantly manifests with cardiomyopathy and/or peripheral neuropathy, but amyloid deposits may be found in other organs or tissues.
Recent Findings
Currently available treatments include transthyretin gene silencers (for hereditary ATTR peripheral neuropathy only) and transthyretin stabilizers (tafamidis for ATTR cardiomyopathy in the USA, and for both hereditary ATTR peripheral neuropathy and ATTR cardiomyopathy in Europe, Japan, Brazil, and some other countries), and liver transplantation. Gene silencers stop the progression of hereditary ATTR peripheral neuropathy in most patients, and transthyretin stabilizers reduce hospitalizations and mortality in patients with ATTR cardiomyopathy. The use of liver transplantation for ATTR has declined with the availability of more effective therapies, and shortage of available allografts. On the horizon are new treatments already in clinical trials including new gene silencers and gene editing agents, new transthyretin stabilizers, and amyloid removal treatments.
Summary
Recently approved treatments for ATTR have changed its natural history, and additional medications may get approved in the near future. Early diagnosis is still essential to improve treatment outcomes. New management strategies may include combinations of gene silencers, transthyretin stabilizers, gene editing, and amyloid removal agents, but the cost may become the limiting factor.
期刊介绍：
This journal aims to review the most important, recently published treatment option advances in the field of neurology. By presenting clear, insightful, balanced contributions by international experts, the journal intends to facilitate worldwide approaches to the treatment of neurologic conditions.
We accomplish this aim by appointing international authorities to serve as Section Editors in key subject areas, such as epilepsy, headache, neurologic ophthalmology and otology, neuromuscular disorders, psychiatric manifestations of neurologic disease, and sleep disorders. Section Editors select topics for which leading experts contribute comprehensive review articles that emphasize new developments and recently published papers of major importance, highlighted by annotated reference lists. We also provide commentaries from well-known neurologists, and an international Editorial Board reviews the annual table of contents, suggests articles of special interest to their country/region, and ensures that topics are current and include emerging research.
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