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

Autor: Dyck PJB; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA., Kincaid JC; Department of Neurology, Indiana University, Indianapolis, Indiana, USA., Wiesman JF; Department of Neurology, New York University, New York, New York, USA., Polydefkis M; Department of Neurology, Johns Hopkins University, Baltimore, Maryland, USA., Litchy WJ; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA., Mauermann ML; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA., Ackermann EJ; Otonomy, Inc., San Diego, California, USA., Guthrie S; Aurora Bio, San Francisco, California, USA., Pollock M; Akcea Therapeutics, Inc., Cambridge, Massachusetts, USA., Jung SW; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA., Baker BF; Ionis Pharmaceuticals, Inc., Carlsbad, California, USA., Dyck PJ; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
Jazyk: angličtina
Zdroj: Muscle & nerve [Muscle Nerve] 2020 Oct; Vol. 62 (4), pp. 502-508. Date of Electronic Publication: 2020 Aug 13.
DOI: 10.1002/mus.27022
Abstrakt: 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.
(© 2020 The Authors. Muscle & Nerve published by Wiley Periodicals LLC.)
Databáze: MEDLINE
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