A Review of Novel Agents and Clinical Considerations in Patients With ATTR Cardiac Amyloidosis.
J Cardiovasc Pharmacol
; 77(5): 544-548, 2021 05 01.
Article
in En
| MEDLINE
| ID: mdl-33657048
ABSTRACT
ABSTRACT Transthyretin (ATTR) amyloidosis is a multisystem disease caused by organ deposition of amyloid fibrils derived from the misfolded transthyretin (TTR) protein. The purpose of this article is to provide an overview of current treatment regimens and summarize important considerations for each agent. A literature search was performed with the PubMed database for articles published through October 2020. Search criteria included therapies available on the market and investigational therapies used for ATTR amyloidosis treatment. Both prospective clinical trials and retrospective studies have been included in this review. Available therapies discussed in this review article are tafamidis, diflunisal, patisiran, and inotersen. Tafamidis is FDA approved for treatment of wild-type ATTR (ATTRwt) and hereditary ATTR (ATTRv) cardiomyopathy, and patisiran and inotersen are FDA approved for ATTRv polyneuropathy. Diflunisal does not have an FDA-labeled indication for amyloidosis but has been studied in ATTRv polyneuropathy and ATTRwt cardiomyopathy. Investigational therapies include a TTR stabilizer, AG10; 2 antifibril agents, PRX004 and doxycycline/tauroursodeoxycholic acid; and 2 gene silencers, vutrisiran and AKCEA-TTR-LRx; and clinical trials are ongoing. ATTR amyloidosis treatment selection is based on subtype and presence of cardiac or neurological manifestations. Additional considerations such as side effects, monitoring, and administration are outlined in this review.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Prealbumin
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Cardiovascular Agents
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Amyloid Neuropathies, Familial
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Mutation
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Cardiomyopathies
Type of study:
Diagnostic_studies
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Observational_studies
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Risk_factors_studies
/
Systematic_reviews
Limits:
Animals
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Humans
Language:
En
Journal:
J Cardiovasc Pharmacol
Year:
2021
Document type:
Article