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Abstract Details

Long-Term Impact of Inotersen on Health-Related Quality-of-Life for Hereditary Transthyretin Amyloidosis with Polyneuropathy: NEURO-TTR Open-Label Extension at Two Years
General Neurology
P6 - Poster Session 6 (12:00 PM-1:00 PM)
6-003
To examine changes in health-related quality-of-life (HRQoL) for patients with hereditary transthyretin amyloidosis (hATTR) with polyneuropathy (hATTR-PN) treated with inotersen for two years in the NEURO-TTR study open-label extension (OLE) phase.
Patients with hATTR, a rare protein misfolding disorder, experience progressive, debilitating polyneuropathy. A randomized, controlled phase 3 trial (NEURO-TTR) demonstrated efficacy in several domains of neuropathic-specific and generic physical HRQoL for patients with hATTR-PN who received inotersen for 66 weeks.
The NEURO-TTR OLE enrolled 135 (of 139) randomized-phase completers who received inotersen (n=85) or placebo (n=50) during the randomized phase. In the OLE phase, all patients received 300 mg inotersen once-weekly. Neuropathic-specific (Norfolk-QOL-Diabetic Neuropathy [DN]) and generic HRQoL (SF-36v2) were assessed at OLE baseline and weeks 26, 78, and 104. Descriptive analyses examined mean changes during the OLE phase for selected instrument domains that showed treatment efficacy during the randomized phase.
For patients receiving either inotersen or placebo during the randomized phase, changes in scores from OLE baseline to week 104 remained stable for Norfolk-QOL-DN activities of daily living (mean change=2.3, 2.0 points, respectively), large fiber neuropathy (1.5, 2.1), and symptoms domains (-0.5, 0.4). Scores were similarly stable during this interval for SF-36v2 physical functioning (mean change=-0.8, -1.6 points, respectively), role-physical (-0.7, -1.0), and bodily pain domains (3.3, 1.6).  Magnitudes of mean differences between the two groups were similar at OLE baseline and week 104, indicating that HRQoL deficits for the randomized placebo group at the end of the randomized phase were undiminished during the OLE phase.
Treatment with inotersen stabilized several domains of neuropathic-specific and generic physical HRQoL for patients with hATTR-PN over two years. The gaps in HRQoL between those previously receiving inotersen versus placebo did not close, indicating the importance of early treatment for maintaining HRQoL in these patients.
Authors/Disclosures
Aaron Yarlas, PhD (Ionis Pharmaceuticals)
PRESENTER 		Dr. Yarlas has received personal compensation for serving as an employee of Ionis Pharmaceuticals. Dr. Yarlas has stock in Ionis Pharmaceuticals.
No disclosure on file
No disclosure on file
Montserrat Vera Llonch Montserrat Vera Llonch has received personal compensation for serving as an employee of Akcea. Montserrat Vera Llonch has received stock or an ownership interest from Akcea.