{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T23:24:46Z","timestamp":1780442686684,"version":"3.54.1"},"reference-count":40,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"ZJLab"},{"name":"Shanghai Municipal Science and Technology Major Project","award":["No.2018SHZDZX01"],"award-info":[{"award-number":["No.2018SHZDZX01"]}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ther Adv Neurol Disord"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:sec><jats:title>Background:<\/jats:title><jats:p> Hereditary transthyretin amyloidosis (ATTRv; v for variant) with polyneuropathy is a rare, progressive, and fatal autosomal dominant disorder. Therapies such as liver transplantation and TTR stabilizations have limitations. Patisiran is a small interfering RNA (siRNA), offering potential as a genetic-level therapy for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). However, evidence on patisiran\u2019s efficacy and safety for ATTRv-PN remains limited. <\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives:<\/jats:title><jats:p> This study aimed to further clarify patisiran\u2019s efficacy and safety for ATTRv-PN by meta-analysis. <\/jats:p><\/jats:sec><jats:sec><jats:title>Design:<\/jats:title><jats:p> Systematic review and meta-analysis. <\/jats:p><\/jats:sec><jats:sec><jats:title>Methods:<\/jats:title><jats:p> After literature searches in PubMed, Ovid MEDLINE, Embase, JBI EBP, Cochrane, and ClinicalTrials.gov databases on 7 June 2024, 11 studies with 503 patients were included and clinical data were extracted. <\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p> Results showed an 88% (95% confidence interval (CI): 81%\u201394%) pooled responsiveness rate. The standardized mean difference of modified Neuropathy Impairment Score plus 7 nerve tests (mNIS\u2009+\u20097) scores was \u22120.18 (95% CI: \u22120.32 to \u22120.03, p-value 0.018) and Norfolk Quality of Life\u2013Diabetic Neuropathy was \u22120.21 (95% CI: \u22120.35 to \u22120.08, p-value 0.002). In total, 413 adverse events (AEs) (84.8%), 158 serious AEs (32.4%), and 37 deaths (7.6%) were recorded. Most of AEs were mild to moderate. No deaths were attributed to patisiran. However, there is no statistically significant improvement in Neuropathy Impairment Scores. <\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion:<\/jats:title><jats:p> In conclusion, patisiran was effective and safe for patients with ATTRv-PN. More large-scale clinical trials and long-term studies are necessary to further validate patisiran\u2019s efficacy and safety. <\/jats:p><\/jats:sec><jats:sec><jats:title>Trial registration:<\/jats:title><jats:p> PROSPERO registration ID: CRD42023428838. <\/jats:p><\/jats:sec>","DOI":"10.1177\/17562864241273079","type":"journal-article","created":{"date-parts":[[2024,9,14]],"date-time":"2024-09-14T02:51:23Z","timestamp":1726282283000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":5,"title":["Efficacy and safety of patisiran for ATTRv-PN: a systematic review and meta-analysis"],"prefix":"10.1177","volume":"17","author":[{"given":"Xinyue","family":"Huang","sequence":"first","affiliation":[{"name":"Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"Rare Disease Center, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"National Center for Neurological Disorders, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5933-0467","authenticated-orcid":false,"given":"Chong","family":"Sun","sequence":"additional","affiliation":[{"name":"Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"Rare Disease Center, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"National Center for Neurological Disorders, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haofeng","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"Rare Disease Center, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"National Center for Neurological Disorders, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9481-1418","authenticated-orcid":false,"given":"Chongbo","family":"Zhao","sequence":"additional","affiliation":[{"name":"Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"Rare Disease Center, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"National Center for Neurological Disorders, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6351-3389","authenticated-orcid":false,"given":"Jie","family":"Lin","sequence":"additional","affiliation":[{"name":"Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, 12# Wulumuqi Zhong Road, Shanghai 200040, China"},{"name":"Rare Disease Center, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China"},{"name":"National Center for Neurological Disorders, Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,9,12]]},"reference":[{"key":"bibr1-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2022.2147636"},{"key":"bibr2-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000001870"},{"key":"bibr3-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000132966.36437.12"},{"key":"bibr4-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s10741-014-9462-7"},{"key":"bibr5-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-10-S1-P19"},{"key":"bibr6-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-013-7051-7"},{"key":"bibr7-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-016-8337-3"},{"key":"bibr8-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-016-8064-9"},{"key":"bibr9-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1208760"},{"key":"bibr10-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1716153"},{"key":"bibr11-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01399-4"},{"key":"bibr12-17562864241273079","unstructured":"Administration UFaD. ONPATTRO (patisiran), https:\/\/www.accessdata.fda.gov\/scripts\/cder\/daf\/index.cfm?event=overview.processApplNo=210922 (2018, accessed 27 August 2023)."},{"key":"bibr13-17562864241273079","unstructured":"European Medicines Agency. Onpattro, https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/onpattro (2018, accessed 27 August 2023)."},{"key":"bibr14-17562864241273079","volume":"372","author":"Page MJ","year":"2021","journal-title":"BMJ"},{"key":"bibr15-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s13760-023-02188-z"},{"key":"bibr16-17562864241273079","first-page":"499","volume":"15","author":"Di Stefano V","year":"2022","journal-title":"Pharmgenomics Pers Med"},{"key":"bibr17-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1111\/ajt.17009"},{"key":"bibr18-17562864241273079","doi-asserted-by":"publisher","DOI":"10.3390\/brainsci11040515"},{"key":"bibr19-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27338"},{"key":"bibr20-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-022-10978-3"},{"key":"bibr21-17562864241273079","doi-asserted-by":"publisher","DOI":"10.3390\/biomedicines11010062"},{"key":"bibr22-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s10072-024-07494-9"},{"key":"bibr23-17562864241273079","volume":"366","author":"Sterne JAC","year":"2019","journal-title":"BMJ"},{"key":"bibr24-17562864241273079","unstructured":"Wells G, Shea BJ, O\u2019Connell D, et al. The Newcastle\u2013Ottawa Scale (NOS) for assessing the quality of nonrandomised studies in meta-analyses, https:\/\/www.ohri.ca\/programs\/clinical_epidemiology\/oxford.asp (2021, accessed 7 August 2023)."},{"key":"bibr25-17562864241273079","doi-asserted-by":"publisher","DOI":"10.13105\/wjma.v5.i4.80"},{"key":"bibr26-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(20)30368-9"},{"key":"bibr27-17562864241273079","doi-asserted-by":"publisher","DOI":"10.3109\/07853890.2015.1068949"},{"key":"bibr28-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2021-327909"},{"key":"bibr29-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1055\/s-0039-1688994"},{"key":"bibr30-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24519"},{"key":"bibr31-17562864241273079","doi-asserted-by":"publisher","DOI":"10.3389\/fgene.2023.1126836"},{"key":"bibr32-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1080\/14737167.2021.1900738"},{"key":"bibr33-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181ea15d4"},{"key":"bibr34-17562864241273079","doi-asserted-by":"publisher","DOI":"10.3109\/13506129.2011.615872"},{"key":"bibr35-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2013.283815"},{"key":"bibr36-17562864241273079","doi-asserted-by":"publisher","DOI":"10.3109\/13506129.2014.997872"},{"key":"bibr37-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3182661eb1"},{"key":"bibr38-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1007\/s40259-023-00577-7"},{"key":"bibr39-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-015-0326-6"},{"key":"bibr40-17562864241273079","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2019.116424"}],"container-title":["Therapeutic Advances in Neurological Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/17562864241273079","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/17562864241273079","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/17562864241273079","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,28]],"date-time":"2025-02-28T17:47:40Z","timestamp":1740764860000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/17562864241273079"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,1]]},"references-count":40,"alternative-id":["10.1177\/17562864241273079"],"URL":"https:\/\/doi.org\/10.1177\/17562864241273079","relation":{},"ISSN":["1756-2864","1756-2864"],"issn-type":[{"value":"1756-2864","type":"print"},{"value":"1756-2864","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,1]]},"article-number":"17562864241273079"}}