帕替西兰治疗 ATTRv-PN 的有效性和安全性：系统回顾和荟萃分析。

IF 5.4 3区材料科学 Q2 CHEMISTRY, PHYSICAL

ACS Applied Energy Materials Pub Date : 2024-09-12 DOI:10.1177/17562864241273079

Xinyue Huang,Chong Sun,Haofeng Chen,Chongbo Zhao,Jie Lin

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引用次数: 0

摘要

背景遗传性转甲状腺素淀粉样变性病（ATTRv；v表示变异型）伴多发性神经病是一种罕见、进行性和致命的常染色体显性遗传疾病。肝移植和TTR稳定剂等疗法都有局限性。帕替西兰是一种小干扰 RNA（siRNA），有望作为遗传性转甲状腺素淀粉样变性伴多发性神经病（ATTRv-PN）的基因水平疗法。本研究旨在通过荟萃分析进一步阐明帕替西兰对 ATTRv-PN 的疗效和安全性。方法2024年6月7日在PubMed、Ovid MEDLINE、Embase、JBI EBP、Cochrane和ClinicalTrials.gov数据库中进行文献检索后，纳入了11项研究，共503名患者，并提取了临床数据。结果结果显示，汇总应答率为88%（95%置信区间（CI）：81%-94%）。改良神经病变损害评分加7项神经测试（mNIS + 7）评分的标准化平均差异为-0.18（95% CI：-0.32至-0.03，P值为0.018），诺福克生活质量-糖尿病神经病变的标准化平均差异为-0.21（95% CI：-0.35至-0.08，P值为0.002）。共记录了 413 例不良事件（84.8%）、158 例严重不良事件（32.4%）和 37 例死亡（7.6%）。大多数不良反应为轻度至中度。帕替西兰没有导致死亡。总之，帕替西兰对 ATTRv-PN 患者有效且安全。有必要进行更多大规模临床试验和长期研究，以进一步验证帕替西兰的有效性和安全性：CRD42023428838。

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Efficacy and safety of patisiran for ATTRv-PN: a systematic review and meta-analysis.

Background 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's efficacy and safety for ATTRv-PN remains limited. Objectives This study aimed to further clarify patisiran's efficacy and safety for ATTRv-PN by meta-analysis. Design Systematic review and meta-analysis. Methods 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. Results Results showed an 88% (95% confidence interval (CI): 81%-94%) pooled responsiveness rate. The standardized mean difference of modified Neuropathy Impairment Score plus 7 nerve tests (mNIS + 7) scores was -0.18 (95% CI: -0.32 to -0.03, p-value 0.018) and Norfolk Quality of Life-Diabetic Neuropathy was -0.21 (95% CI: -0.35 to -0.08, 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. Conclusion 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's efficacy and safety. Trial registration PROSPERO registration ID: CRD42023428838.

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来源期刊

ACS Applied Energy Materials Materials Science-Materials Chemistry

CiteScore

10.30

自引率

6.20%

发文量

1368

期刊介绍： ACS Applied Energy Materials is an interdisciplinary journal publishing original research covering all aspects of materials, engineering, chemistry, physics and biology relevant to energy conversion and storage. The journal is devoted to reports of new and original experimental and theoretical research of an applied nature that integrate knowledge in the areas of materials, engineering, physics, bioscience, and chemistry into important energy applications.