ArticleNeurology and therapy2026
Efficacy and Safety of Vutrisiran in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: Analysis of the East Asian Subpopulation from HELIOS-A.
Article in Neurology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03759379 (HELIOS-A), which is not on this 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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Who cites it
1 citing paper in PubMed.
- Cardiac Amyloidosis in Asia: What We Know and Don't Know.JACC. Asia · 2026Article
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PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
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12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionIn the phase 3 HELIOS-A study (NCT03759379), vutrisiran significantly improved a range of disease-related endpoints compared with an external placebo in patients with hereditary transthyretin amyloidosis (ATTRv) with polyneuropathy (ATTRv-PN). This post hoc analysis examined the effects of vutrisiran in East Asian participants of HELIOS-A.
methodsHELIOS-A was a global, open-label, phase 3 study in which patients with ATTRv-PN were randomized 3:1 to receive subcutaneous vutrisiran 25 mg every 3 months or intravenous patisiran 0.3 mg/kg every 3 weeks for 18 months. The placebo group of the APOLLO study was used as an external placebo control. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score + 7 (mNIS + 7) score versus placebo at month 9; secondary endpoints included change from baseline in: Norfolk Quality of Life-Diabetic Neuropathy questionnaire score and 10-meter walk test speed at months 9 and 18, mNIS + 7 score, modified body mass index and Rasch-built Overall Disability Scale score at month 18, and serum transthyretin level through month 18.
resultsOf the 122 patients receiving vutrisiran in HELIOS-A and 77 patients receiving placebo in APOLLO, 33 were East Asian (vutrisiran: 12, placebo: 21) and had similar baseline characteristics to the overall study cohorts. All outcomes, including the primary endpoint of change from baseline in mNIS + 7 score, showed changes that were consistent in direction and magnitude with the changes seen in the overall HELIOS-A analysis set. Adverse events (AEs) were broadly similar between the East Asian subpopulation and the overall HELIOS-A population; compared with the APOLLO placebo group, fewer patients died or experienced treatment-related AEs and serious AEs.
conclusionAlthough the number of East Asian patients in HELIOS-A was small, this post hoc analysis showed similar outcomes to those in the overall study populations, indicating that vutrisiran is effective and well tolerated in East Asian patients.
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