Premium
Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
Author(s) -
Dyck P. James B.,
Coelho Teresa,
Waddington Cruz Marcia,
Brannagan Thomas H.,
Khella Sami,
Karam Chafic,
Berk John L.,
Polydefkis Michael J.,
Kincaid John C.,
Wiesman Janice F.,
Litchy William J.,
Mauermann Michelle L.,
Ackermann Elizabeth J.,
Baker Brenda F.,
Jung Shiangtung W.,
Guthrie Spencer,
Pollock Michael,
Dyck Peter J.
Publication year - 2020
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.27023
Subject(s) - medicine , amyloidosis , transthyretin , neuropathic pain , polyneuropathy , peripheral neuropathy , placebo , physical therapy , gastroenterology , diabetes mellitus , anesthesia , pathology , endocrinology , alternative medicine
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 total NSC score. There were also improvements in the subdomains of muscle weakness, sensory, pain, and autonomic symptoms, and for various individual items. Discussion 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.