Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2024 Sep 2:15:1465747.
doi: 10.3389/fneur.2024.1465747. eCollection 2024.

Assessing the effectiveness and safety of Patisiran and Vutrisiran in ATTRv amyloidosis with polyneuropathy: a systematic review

Mohammad Amin Karimi #  1 , Fatemeh Esmaeilpour Moallem #  2 , Mohammad Sadra Gholami Chahkand  2 , Eftekhar Azarm  3 , Mohammad Javad Emami Kazemabad  4 , Parisa Alsadat Dadkhah  3
Affiliations

Assessing the effectiveness and safety of Patisiran and Vutrisiran in ATTRv amyloidosis with polyneuropathy: a systematic review

Mohammad Amin Karimi et al. Front Neurol. .

Abstract

Background: Hereditary transthyretin (ATTRv) amyloidosis, a multifaceted disorder affecting multiple systems, substantially diminishes patients' physical capabilities and overall quality of life. Patisiran and Vutrisiran, two Ribonucleic acid (RNA) interference therapies, target reducing both pathogenic and wild-type transthyretin (TTR) protein levels. This systematic review assesses the effectiveness and safety of these treatments in managing ATTRv.

Methods: A comprehensive, thorough literature search across databases including Embase, PubMed, Web of Science, Cochrane Central, and Google Scholar yielded 858 studies. Following removing duplicate and irrelevant articles, 676 distinct studies underwent review. These studies, conducted on a global scale, encompassed a range of methodologies, including clinical trials and indirect treatment comparisons.

Results: Ten studies, spanning a total population of 756 patients, were selected for in-depth analysis. Patisiran and Vutrisiran consistently demonstrated significant improvements in primary and secondary endpoints related to neuropathy, quality of life, and cardiac function. Both medications were well-tolerated, with primarily mild to moderate adverse events. Indirect treatment comparison studies indicated Vutrisiran's superiority over Tafamidis in treating ATTRv amyloidosis.

Conclusion: This systematic review recommends using Patisiran and Vutrisiran to treat ATTRv amyloidosis. The findings suggest that these RNA interference therapies improve neuropathy, quality of life, and cardiac symptoms. The results indicate sustained benefits over prolonged treatment, with satisfactory safety profiles. However, potential biases, conflicts of interest in the studies, and limited follow-up periods in some trials necessitate cautious interpretation. Future research should address these limitations and provide more robust evidence for the long-term efficacy and safety of Patisiran and Vutrisiran in ATTRv treatment.

Keywords: Patisiran; Vutrisiran; cardiomyopathy; clinical trials; hereditary amyloidosis; polyneuropathy; transthyretin-related amyloidosis.

PubMed Disclaimer

Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Similar articles

See all similar articles

Cited by

  • Patisiran in ATTRv amyloidosis with polyneuropathy: "PatisiranItaly" multicenter observational study.
    Di Stefano V, Guaraldi P, Romano A, Antonini G, Barilaro A, Briani C, Burattini M, Cani I, Carlini G, Ceccanti M, Cianci V, Cortelli P, Currò Dossi M, Di Lisi D, Di Muzio A, Falzone Y, Filosto M, Gasverde S, Gemelli C, Gentile L, Goglia M, Leonardi L, Longhi S, Lotti A, Manganelli F, Mazzeo A, Milella G, Novo G, Fenu S, Palumbo G, Petrelli C, Poli L, Pradotto LG, Russo M, Salvalaggio A, Sciarrone MA, Sellitti L, Tagliapietra M, Tozza S, Turri M, Verriello L, Vitali F, Brighina F, Luigetti M. Di Stefano V, et al. J Neurol. 2025 Feb 15;272(3):209. doi: 10.1007/s00415-025-12950-3. J Neurol. 2025. PMID: 39954098 Free PMC article.
  • Amyloids and the Heart: An Update.
    Triposkiadis F, Briasoulis A, Xanthopoulos A. Triposkiadis F, et al. J Clin Med. 2024 Nov 27;13(23):7210. doi: 10.3390/jcm13237210. J Clin Med. 2024. PMID: 39685666 Free PMC article. Review.

References

    1. Adams D, Koike H, Slama M, Coelho T. Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease. Nat Rev Neurol. (2019) 15:387–404. doi: 10.1038/s41582-019-0210-4, PMID: - DOI - PubMed
    1. Manganelli F, Fabrizi GM, Luigetti M, Mandich P, Mazzeo A, Pareyson D. Hereditary transthyretin amyloidosis overview. Neurol Sci. (2022) 43:595–604. doi: 10.1007/s10072-020-04889-2 - DOI - PMC - PubMed
    1. Koike H, Misu K-i, Ikeda S-i, Ando Y, Nakazato M, Ando E, et al. . Type I (transthyretin met 30) familial amyloid polyneuropathy in Japan: early-vs late-onset form. Arch Neurol. (2002) 59:1771–6. doi: 10.1001/archneur.59.11.1771 - DOI - PubMed
    1. Carroll A, Dyck PJ, de Carvalho M, Kennerson M, Reilly MM, Kiernan MC, et al. . Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis. J Neurol Neurosurg Psychiatry. (2022) 93:668–78. doi: 10.1136/jnnp-2021-327909, PMID: - DOI - PMC - PubMed
    1. Tozza S, Severi D, Spina E, Iovino A, Aruta F, Ruggiero L, et al. . The neuropathy in hereditary transthyretin amyloidosis: a narrative review. J Peripher Nerv Syst. (2021) 26:155–9. doi: 10.1111/jns.12451, PMID: - DOI - PMC - PubMed

Publication types

LinkOut - more resources