Efficacy and safety of patisiran for ATTRv-PN: a systematic review and meta-analysis
| Autor: | Xinyue Huang, Chong Sun, Haofeng Chen, Chongbo Zhao, Jie Lin |
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| Jazyk: | angličtina |
| Rok vydání: | 2024 |
| Předmět: | |
| Zdroj: | Therapeutic Advances in Neurological Disorders, Vol 17 (2024) |
| Druh dokumentu: | article |
| ISSN: | 1756-2864 17562864 |
| DOI: | 10.1177/17562864241273079 |
| Popis: | 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. |
| Databáze: | Directory of Open Access Journals |
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