SynthesisTherapeutic advances in cardiovascular disease
Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials.
Synthesis in Therapeutic advances in cardiovascular disease. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
- Comments on "Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials".Therapeutic advances in cardiovascular diseaseArticle
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Authors and funding
10 authors.
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Abstract
backgroundHereditary transthyretin amyloidosis (hATTR) is caused by mutations in the transthyretin (TTR) gene, which lead to the aggregation of misfolded TTR protein and amyloid accumulation in the peripheral nerves, heart, and gastrointestinal tract. Recently, RNA therapeutics, including small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs), have been approved for treating patients with hATTR.
objectivesTo assess the neurological efficacy and safety of RNA therapeutics in hATTR patients.
designSystematic review and meta-analysis. DATA SOURCES AND
methodsA systematic literature search was conducted on PubMed, Cochrane, and ClinicalTrials.gov from inception to August 14, 2024. Outcomes included changes from baseline in the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) score and the modified Neuropathy Impairment Score +7 (mNIS + 7), modified body mass index (mBMI), adverse effects, serious adverse events, and all-cause mortality.
resultsOur study included four RCTs with 842 patients (568 in the RNA therapeutics group and 274 in the placebo group). RNA therapeutics significantly improved Norfolk QoL-DN (mean difference (MD), -18.79; 95% CI, -22.32 to -15.25;
conclusionRNA therapeutics are effective and well-tolerated in patients with hATTR, significantly improving quality of life and the progression of neurological impairment. siRNAs demonstrate better outcomes compared to ASOs.
trial registrationPROSPERO (CRD42024568346).
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