Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis.

ABSTRACT:

Introduction

Hereditary transthyretin-mediated amyloidosis (hATTR) manifests as multisystem dysfunction, including progressive polyneuropathy. Inotersen, an antisense oligonucleotide, improved the course of neuropathic impairment in patients with hATTR in the pivotal NEURO-TTR study (NCT01737398). To determine inotersen's impact on symptoms and patients' neuropathy experience, we performed a post hoc analysis of the Neuropathy Symptoms and Change (NSC) score.

Methods

Stage 1 or 2 hATTR patients were randomized to receive weekly subcutaneous inotersen or placebo for 65 weeks. NSC score was assessed at baseline and 35 and 66 weeks.

Results

At 66 weeks, inotersen-treated patients had symptom stabilization as compared with worsening in patients receiving placebo, based on

SUBMITTER: Dyck PJB 

PROVIDER: S-EPMC7540369 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature