Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
Université Paris-Saclay · Assistance Publique – Hôpitaux de Paris · +22 more institutions
Abstract
The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy.
HELIOS-A was a phase 3, global, open-label study comparing the efficacy and safety of vutrisiran with an external placebo group (APOLLO study). Patients were randomized 3:1 to subcutaneous vutrisiran 25 mg every 3 months (Q3M) or intravenous patisiran 0.3 mg/kg every 3 weeks (Q3W) for 18 months.
Citation impact
- FWCI
- 27.89
- Percentile
- 100%
- References
- 31
Authors
17- DADavid AdamsCorresponding
Université Paris-Saclay, Assistance Publique – Hôpitaux de Paris, Bicêtre Hospital
- ITIvailo Tournev
New Bulgarian University, Alexandrovska Hospital
- MTMark Taylor
The University of Sydney, Westmead Hospital
- TCTeresa Coelho
Hospital de Santo António, Centro Hospitalar do Porto
- VPViolaine Planté‐Bordeneuve
Université Paris-Est Créteil, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique – Hôpitaux de Paris
Topics & keywords
- Transthyretin
- Medicine
- Amyloidosis
- Polyneuropathy
- Randomized controlled trial
- Clinical trial
- Internal medicine
- Amyloid polyneuropathy