Main idea: Inotersen treatment is efficient in neuropathy symptoms reduction in patients with hereditary transthyretin-mediated amyloidosis.
Abstract: 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. 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. 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. Inotersen treatment stabilized neuropathy symptoms, including autonomic symptoms, in patients with hATTR according to NSC score. Thus, the NSC may be an effective measure to assess neuropathy progression and patients” neuropathy experience in clinical practice.