MNIS+7 and lower limb function in inotersen treatment of hereditary transthyretin-mediated amyloidosis.

ABSTRACT: INTRODUCTION:Inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, demonstrated significant benefit versus placebo in the modified Neuropathy Impairment Score (NIS)?+7 neurophysiologic tests (mNIS+7) in patients with hereditary TTR-mediated amyloidosis (hATTR) with polyneuropathy. This analysis assessed the mNIS+7 components by anatomic location and the lower limb function (LLF) test. METHODS:Adults with hATTR in the NEURO-TTR trial (NCT01737398) were randomly assigned to receive weekly doses of subcutaneous inotersen 300?mg or placebo for 65?weeks. The mNIS+7 and LLF were assessed at 35 and 66?weeks. RESULTS:All major mNIS+7 components (muscle weakness, muscle stretch reflexes, sensation) and the LLF showed significant efficacy in patients receiving inotersen versus placebo; however, NIS-reflexes (upper limb), touch pressure (upper and lower limbs), and heart rate during deep breathing did not show significant effects. DISCUSSION:The results of this analysis reinforce the beneficial effect of inotersen on slowing neuropathy progression in patients with hATTR polyneuropathy.

SUBMITTER: Dyck PJB 

PROVIDER: S-EPMC7540053 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature