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Update on Amyloid Polyneuropathy and Treatment

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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.

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Availability of Data and Materials

No datasets were generated or analyzed during the current study.

Abbreviations

AL:

Light-chain amyloidosis

ASO:

Antisense oligonucleotide

ATTR:

Transthyretin amyloidosis

ATTR-CM:

Transthyretin amyloid cardiomyopathy

CNS:

Central nervous system

CTS:

Carpal tunnel syndrome

FAP:

Familial amyloid polyneuropathy

GalNAc:

N-Acetylgalactosamine

hATTR:

Hereditary transthyretin amyloidosis

hATTR-CM:

Hereditary transthyretin amyloid cardiomyopathy

hATTR-PN:

Hereditary transthyretin amyloid peripheral neuropathy

mRNA:

Messenger RNA

Norfolk QOL-DN:

Norfolk Quality of Life-Diabetic Neuropathy

OLMA:

Oculoleptomeningeal amyloidosis

PND:

Polyneuropathy Disability scale

RISC:

RNA-induced silencing complex

siRNA:

Small interfering RNA

TTR:

Transthyretin amyloidosis

wtATTR:

Wild-type transthyretin amyloidosis

References and Recommended Reading

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Authors and Affiliations

  1. Department of Neurology, Yale University, New Haven, CT, USA

    Sasha A. Živković MD, PhD

  2. Department of Neurology, Geisinger Medical Center, Danville, PA, USA

    J. David Avila MD

  3. Section of Cardiovascular Medicine, Yale University, New Haven, CT, USA

    Cesia Gallegos-Kattan MD, MHS

  4. Department of Neurology, University of Colorado Anschutz, Aurora, CO, USA

    Dianna Quan MD

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Correspondence to Sasha A. Živković MD, PhD.

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Sasha A. Živković — Consulting and/or advisory boards with Alnylam Pharmaceuticals, Astra Zeneca, Argenx, and Takeda. J. David Avila — Speaker for Alnylam, Argenx, Alexion, and UCB; consulting for Alnylam Pharmaceuticals and Alexion. Cesia Gallegos-Kattan — Advisory board for Alnylam Pharmaceuticals. Dianna Quan — Research funding, speaking fees, and travel expenses from Alnylam Pharmaceuticals; research funding from Pfizer, Ionis, Cytokinetics, Momenta/Janssen, Viela Bio, and Avidity Biosciences.

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Živković, S.A., Avila, J.D., Gallegos-Kattan, C. et al. Update on Amyloid Polyneuropathy and Treatment. Curr Treat Options Neurol 26, 51–66 (2024). https://doi.org/10.1007/s11940-024-00780-z

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