Patisiran in ATTRv amyloidosis with polyneuropathy: “PatisiranItaly” multicenter observational study
Background Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the TTR gene, leading to misfolded monomers that aggregate, for...
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| Published in | Journal of neurology Vol. 272; no. 3; p. 209 |
|---|---|
| Main Authors | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Berlin/Heidelberg
Springer Berlin Heidelberg
01.03.2025
Springer Nature B.V |
| Subjects | |
| Online Access | Get full text |
| ISSN | 0340-5354 1432-1459 1432-1459 |
| DOI | 10.1007/s00415-025-12950-3 |
Cover
| Abstract | Background
Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the
TTR
gene, leading to misfolded monomers that aggregate, forming amyloid fibrils. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, designed to enter hepatocytes and selectively target TTR mRNA to reduce both variant TTR and wild-type TTR (wt). This study presents a multicenter, real-life experience of patisiran’s effectiveness and safety in ATTRv-PN.
Methods
We enrolled genetically confirmed ATTRv-PN patients from 29 specialized Italian centers. All subjects underwent neurological assessments, including familial amyloid polyneuropathy (FAP) staging, the Neuropathy Impairment Score (NIS), quality-of-life assessment using the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, and the Compound Autonomic Dysfunction Test (CADT). Additional assessments included baseline and follow-up measures of serum NT-proBNP and interventricular septal thickness.
Results
A total of 181 ATTRv patients (69% male) were enrolled. Neurological onset was reported in 60.2% of cases. At baseline, 83.4% of patients exhibited multisystemic involvement, while only 16.6% presented isolated polyneuropathy. For approximately 70% of patients, patisiran was the first treatment; the remainder transitioned from tafamidis or inotersen. Following treatment, most patients demonstrated stabilization of neuropathy progression, regardless of baseline disease severity or genotype. The treatment was well-tolerated, with 90% of patients reporting no adverse events.
Conclusion
Patisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran. |
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| AbstractList | BackgroundHereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the TTR gene, leading to misfolded monomers that aggregate, forming amyloid fibrils. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, designed to enter hepatocytes and selectively target TTR mRNA to reduce both variant TTR and wild-type TTR (wt). This study presents a multicenter, real-life experience of patisiran’s effectiveness and safety in ATTRv-PN.MethodsWe enrolled genetically confirmed ATTRv-PN patients from 29 specialized Italian centers. All subjects underwent neurological assessments, including familial amyloid polyneuropathy (FAP) staging, the Neuropathy Impairment Score (NIS), quality-of-life assessment using the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, and the Compound Autonomic Dysfunction Test (CADT). Additional assessments included baseline and follow-up measures of serum NT-proBNP and interventricular septal thickness.ResultsA total of 181 ATTRv patients (69% male) were enrolled. Neurological onset was reported in 60.2% of cases. At baseline, 83.4% of patients exhibited multisystemic involvement, while only 16.6% presented isolated polyneuropathy. For approximately 70% of patients, patisiran was the first treatment; the remainder transitioned from tafamidis or inotersen. Following treatment, most patients demonstrated stabilization of neuropathy progression, regardless of baseline disease severity or genotype. The treatment was well-tolerated, with 90% of patients reporting no adverse events.ConclusionPatisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran. Background Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the TTR gene, leading to misfolded monomers that aggregate, forming amyloid fibrils. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, designed to enter hepatocytes and selectively target TTR mRNA to reduce both variant TTR and wild-type TTR (wt). This study presents a multicenter, real-life experience of patisiran’s effectiveness and safety in ATTRv-PN. Methods We enrolled genetically confirmed ATTRv-PN patients from 29 specialized Italian centers. All subjects underwent neurological assessments, including familial amyloid polyneuropathy (FAP) staging, the Neuropathy Impairment Score (NIS), quality-of-life assessment using the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, and the Compound Autonomic Dysfunction Test (CADT). Additional assessments included baseline and follow-up measures of serum NT-proBNP and interventricular septal thickness. Results A total of 181 ATTRv patients (69% male) were enrolled. Neurological onset was reported in 60.2% of cases. At baseline, 83.4% of patients exhibited multisystemic involvement, while only 16.6% presented isolated polyneuropathy. For approximately 70% of patients, patisiran was the first treatment; the remainder transitioned from tafamidis or inotersen. Following treatment, most patients demonstrated stabilization of neuropathy progression, regardless of baseline disease severity or genotype. The treatment was well-tolerated, with 90% of patients reporting no adverse events. Conclusion Patisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran. Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the TTR gene, leading to misfolded monomers that aggregate, forming amyloid fibrils. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, designed to enter hepatocytes and selectively target TTR mRNA to reduce both variant TTR and wild-type TTR (wt). This study presents a multicenter, real-life experience of patisiran's effectiveness and safety in ATTRv-PN.BACKGROUNDHereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the TTR gene, leading to misfolded monomers that aggregate, forming amyloid fibrils. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, designed to enter hepatocytes and selectively target TTR mRNA to reduce both variant TTR and wild-type TTR (wt). This study presents a multicenter, real-life experience of patisiran's effectiveness and safety in ATTRv-PN.We enrolled genetically confirmed ATTRv-PN patients from 29 specialized Italian centers. All subjects underwent neurological assessments, including familial amyloid polyneuropathy (FAP) staging, the Neuropathy Impairment Score (NIS), quality-of-life assessment using the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, and the Compound Autonomic Dysfunction Test (CADT). Additional assessments included baseline and follow-up measures of serum NT-proBNP and interventricular septal thickness.METHODSWe enrolled genetically confirmed ATTRv-PN patients from 29 specialized Italian centers. All subjects underwent neurological assessments, including familial amyloid polyneuropathy (FAP) staging, the Neuropathy Impairment Score (NIS), quality-of-life assessment using the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, and the Compound Autonomic Dysfunction Test (CADT). Additional assessments included baseline and follow-up measures of serum NT-proBNP and interventricular septal thickness.A total of 181 ATTRv patients (69% male) were enrolled. Neurological onset was reported in 60.2% of cases. At baseline, 83.4% of patients exhibited multisystemic involvement, while only 16.6% presented isolated polyneuropathy. For approximately 70% of patients, patisiran was the first treatment; the remainder transitioned from tafamidis or inotersen. Following treatment, most patients demonstrated stabilization of neuropathy progression, regardless of baseline disease severity or genotype. The treatment was well-tolerated, with 90% of patients reporting no adverse events.RESULTSA total of 181 ATTRv patients (69% male) were enrolled. Neurological onset was reported in 60.2% of cases. At baseline, 83.4% of patients exhibited multisystemic involvement, while only 16.6% presented isolated polyneuropathy. For approximately 70% of patients, patisiran was the first treatment; the remainder transitioned from tafamidis or inotersen. Following treatment, most patients demonstrated stabilization of neuropathy progression, regardless of baseline disease severity or genotype. The treatment was well-tolerated, with 90% of patients reporting no adverse events.Patisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran.CONCLUSIONPatisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran. Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting sensorimotor nerves, and various organs. It is caused by mutations in the TTR gene, leading to misfolded monomers that aggregate, forming amyloid fibrils. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, designed to enter hepatocytes and selectively target TTR mRNA to reduce both variant TTR and wild-type TTR (wt). This study presents a multicenter, real-life experience of patisiran's effectiveness and safety in ATTRv-PN. We enrolled genetically confirmed ATTRv-PN patients from 29 specialized Italian centers. All subjects underwent neurological assessments, including familial amyloid polyneuropathy (FAP) staging, the Neuropathy Impairment Score (NIS), quality-of-life assessment using the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, and the Compound Autonomic Dysfunction Test (CADT). Additional assessments included baseline and follow-up measures of serum NT-proBNP and interventricular septal thickness. A total of 181 ATTRv patients (69% male) were enrolled. Neurological onset was reported in 60.2% of cases. At baseline, 83.4% of patients exhibited multisystemic involvement, while only 16.6% presented isolated polyneuropathy. For approximately 70% of patients, patisiran was the first treatment; the remainder transitioned from tafamidis or inotersen. Following treatment, most patients demonstrated stabilization of neuropathy progression, regardless of baseline disease severity or genotype. The treatment was well-tolerated, with 90% of patients reporting no adverse events. Patisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran. |
| ArticleNumber | 209 |
| Author | Romano, Angela Gentile, Luca Luigetti, Marco Petrelli, Cristina Gasverde, Sabrina Mazzeo, Anna Palumbo, Giovanni Fenu, Silvia Guaraldi, Pietro Ceccanti, Marco Cani, Ilaria Brighina, Filippo Cianci, Vittoria Milella, Giammarco Burattini, Marco Poli, Loris Filosto, Massimiliano Salvalaggio, Alessandro Antonini, Giovanni Sellitti, Luigi Carlini, Giulia Longhi, Simone Lotti, Antonio Briani, Chiara Falzone, Yuri Manganelli, Fiore Tozza, Stefano Di Lisi, Daniela Leonardi, Luca Cortelli, Pietro Di Stefano, Vincenzo Gemelli, Chiara Vitali, Francesca Barilaro, Alessandro Russo, Massimo Sciarrone, Maria Ausilia Goglia, Mariangela Currò Dossi, Marco Di Muzio, Antonio Pradotto, Luca Guglielmo Verriello, Lorenzo Novo, Giuseppina Tagliapietra, Matteo Turri, Mara |
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| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/39954098$$D View this record in MEDLINE/PubMed |
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| Cites_doi | 10.1007/s10072-016-2767-7 10.1016/S1474-4422(23)00334-4 10.1007/s10741-021-10080-2 10.2147/PGPM.S359851 10.1007/s10072-023-06977-5 10.1186/s13023-020-01623-1 10.1080/13506129.2020.1730790 10.1080/13506129.2020.1794807 10.3390/biomedicines11010062 10.1016/S1474-4422(20)30458-0 10.1016/S1474-4422(20)30368-9 10.1001/jamaneurol.2024.4631 10.1056/NEJMoa1716153 10.1007/s40120-020-00210-7 10.1212/WNL.0000000000001870 10.3390/brainsci11040515 10.1007/s10072-024-07717-z 10.1007/s10072-024-07494-9 10.1016/S1474-4422(20)30397-5 10.3390/jcm13164914 10.1038/s41582-019-0210-4 10.1161/CIRCULATIONAHA.118.035831 10.3233/JND-150091 10.1007/s00415-019-09688-0 10.3389/fneur.2024.1415851 10.3390/brainsci10120952 10.3390/brainsci11050545 10.1212/01.wnl.0000429338.33391.87 10.3389/fneur.2024.1465747 10.1080/13506129.2018.1564903 10.1056/NEJMoa1716793 10.1080/13506129.2020.1773425 |
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| Keywords | Hereditary transthyretin amyloidosis Patisiran RNA interference Multicenter study ATTRv-PN Real-world data |
| Language | English |
| License | 2025. The Author(s). Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. cc-by |
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| References | M Russo (12950_CR9) 2021; 11 M Russo (12950_CR25) 2020; 27 D Adams (12950_CR16) 2018; 379 L Obici (12950_CR17) 2020; 27 F Vitali (12950_CR29) 2023; 44 MA Karimi (12950_CR15) 2024; 15 G Urbinati (12950_CR18) 2024; 15 M Russo (12950_CR13) 2020; 27 V Di Stefano (12950_CR20) 2022; 15 D Adams (12950_CR28) 2015; 85 D Adams (12950_CR7) 2021; 268 D Di Lisi (12950_CR22) 2024; 13 D Adams (12950_CR1) 2023; 22 D Adams (12950_CR4) 2019; 15 H Koike (12950_CR2) 2020; 9 12950_CR26 IS Merkies (12950_CR10) 2013; 80 V Di Stefano (12950_CR24) 2022; 11 M Vera-Llonch (12950_CR6) 2021; 16 MD Benson (12950_CR11) 2018; 379 N Ohashi (12950_CR3) 2019; 26 D Adams (12950_CR27) 2025 M Russo (12950_CR8) 2020; 10 C Stancanelli (12950_CR30) 2017; 38 JN Nativi-Nicolau (12950_CR5) 2022; 27 A Mazzeo (12950_CR12) 2015; 2 L Gentile (12950_CR23) 2021; 11 SD Solomon (12950_CR21) 2019; 139 M Luigetti (12950_CR14) 2021; 20 12950_CR19 |
| References_xml | – volume: 38 start-page: 525 issue: 3 year: 2017 ident: 12950_CR30 publication-title: Neurol Sci doi: 10.1007/s10072-016-2767-7 – volume: 22 start-page: 1061 issue: 11 year: 2023 ident: 12950_CR1 publication-title: Lancet Neurol doi: 10.1016/S1474-4422(23)00334-4 – volume: 27 start-page: 785 issue: 3 year: 2022 ident: 12950_CR5 publication-title: Heart Fail Rev doi: 10.1007/s10741-021-10080-2 – volume: 15 start-page: 499 year: 2022 ident: 12950_CR20 publication-title: Pharmgenomics Pers Med doi: 10.2147/PGPM.S359851 – volume: 44 start-page: 4569 issue: 12 year: 2023 ident: 12950_CR29 publication-title: Neurol Sci doi: 10.1007/s10072-023-06977-5 – volume: 16 start-page: 25 issue: 1 year: 2021 ident: 12950_CR6 publication-title: Orphanet J Rare Dis doi: 10.1186/s13023-020-01623-1 – volume: 27 start-page: 153 issue: 3 year: 2020 ident: 12950_CR17 publication-title: Amyloid doi: 10.1080/13506129.2020.1730790 – volume: 27 start-page: 259 issue: 4 year: 2020 ident: 12950_CR13 publication-title: Amyloid doi: 10.1080/13506129.2020.1794807 – volume: 11 start-page: 62 issue: 1 year: 2022 ident: 12950_CR24 publication-title: Biomedicines doi: 10.3390/biomedicines11010062 – ident: 12950_CR19 doi: 10.1016/S1474-4422(20)30458-0 10.1016/S1474-4422(20)30368-9 – year: 2025 ident: 12950_CR27 publication-title: JAMA Neurol doi: 10.1001/jamaneurol.2024.4631 – volume: 379 start-page: 11 issue: 1 year: 2018 ident: 12950_CR16 publication-title: N Engl J Med doi: 10.1056/NEJMoa1716153 – volume: 9 start-page: 317 issue: 2 year: 2020 ident: 12950_CR2 publication-title: Neurol Ther doi: 10.1007/s40120-020-00210-7 – volume: 85 start-page: 675 issue: 8 year: 2015 ident: 12950_CR28 publication-title: Neurology doi: 10.1212/WNL.0000000000001870 – volume: 11 start-page: 515 issue: 4 year: 2021 ident: 12950_CR23 publication-title: Brain Sci doi: 10.3390/brainsci11040515 – ident: 12950_CR26 doi: 10.1007/s10072-024-07717-z 10.1007/s10072-024-07494-9 – volume: 20 start-page: 21 issue: 1 year: 2021 ident: 12950_CR14 publication-title: Lancet Neurol doi: 10.1016/S1474-4422(20)30397-5 – volume: 13 start-page: 4914 issue: 16 year: 2024 ident: 12950_CR22 publication-title: J Clin Med doi: 10.3390/jcm13164914 – volume: 15 start-page: 387 issue: 7 year: 2019 ident: 12950_CR4 publication-title: Nat Rev Neurol doi: 10.1038/s41582-019-0210-4 – volume: 139 start-page: 431 issue: 4 year: 2019 ident: 12950_CR21 publication-title: Circulation doi: 10.1161/CIRCULATIONAHA.118.035831 – volume: 2 start-page: S39 issue: s2 year: 2015 ident: 12950_CR12 publication-title: J Neuromuscul Dis doi: 10.3233/JND-150091 – volume: 268 start-page: 2109 issue: 6 year: 2021 ident: 12950_CR7 publication-title: J Neurol doi: 10.1007/s00415-019-09688-0 – volume: 15 start-page: 1415851 year: 2024 ident: 12950_CR18 publication-title: Front Neurol doi: 10.3389/fneur.2024.1415851 – volume: 10 start-page: 952 issue: 12 year: 2020 ident: 12950_CR8 publication-title: Brain Sci doi: 10.3390/brainsci10120952 – volume: 11 start-page: 545 issue: 5 year: 2021 ident: 12950_CR9 publication-title: Brain Sci doi: 10.3390/brainsci11050545 – volume: 80 start-page: 1444 issue: 15 year: 2013 ident: 12950_CR10 publication-title: Neurology doi: 10.1212/01.wnl.0000429338.33391.87 – volume: 15 start-page: 1465747 year: 2024 ident: 12950_CR15 publication-title: Front Neurol doi: 10.3389/fneur.2024.1465747 – volume: 26 start-page: 15 issue: 1 year: 2019 ident: 12950_CR3 publication-title: Amyloid doi: 10.1080/13506129.2018.1564903 – volume: 379 start-page: 22 issue: 1 year: 2018 ident: 12950_CR11 publication-title: N Engl J Med doi: 10.1056/NEJMoa1716793 – volume: 27 start-page: 279 issue: 4 year: 2020 ident: 12950_CR25 publication-title: Amyloid doi: 10.1080/13506129.2020.1773425 |
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Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease,... Hereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease, affecting... BackgroundHereditary amyloid transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, inherited, multisystemic, progressive adult-onset disease,... |
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| SubjectTerms | Adult Aged Amyloid Neuropathies, Familial - complications Amyloid Neuropathies, Familial - drug therapy Amyloid Neuropathies, Familial - genetics Amyloidosis Autonomic nervous system Diabetes mellitus Diabetic neuropathy Double-stranded RNA Female Fibrils Genotypes Hepatocytes Humans Italy Male Medicine Medicine & Public Health Middle Aged mRNA Nanoparticles Nerves Neurology Neuroradiology Neurosciences Observational studies Original Communication Polyneuropathies - drug therapy Polyneuropathy Prealbumin - genetics Quality of Life RNA, Small Interfering - therapeutic use Sensorimotor system Transthyretin Treatment Outcome |
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| Title | Patisiran in ATTRv amyloidosis with polyneuropathy: “PatisiranItaly” multicenter observational study |
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