OBJECTIVES: Hereditary transthyretin (hATTR) amyloidosis is a systemic disease in which misfolded protein deposits (amyloids) gather in the peripheral nervous system and/or cardiac tissues, as well as tissues in other organs and systems, producing clinical manifestations such as polyneuropathy, cardiomyopathy, and gastrointestinal and visual problems. hATTR amyloidosis is rare, progressive, degenerative, and fatal, and lacks established treatments. This analysis examined the real world impact of this disease and treatment with inotersen on patients’ everyday activities.

METHODS: Data were from a multinational, double-blind, placebo-controlled trial of inotersen for 172 adults with hATTR amyloidosis (NCT01737398). The SF-36v2® Health Survey (SF-36v2) was administered to patients at baseline and 15 months post-baseline. Descriptive analysis examined treatment differences in patients’ item-level responses to contextually-meaningful items on the SF-36v2 physical functioning, role-physical, pain, social functioning, and role-emotional scales. The percentage of patients indicating they were ‘limited a lot’ in physical activities; whose physical or emotional health interfered with work and other activities ‘most of the time’ or ‘all of the time’; and whose health or pain interfered with social activities ‘extremely’ or ‘quite a bit’ were tallied at baseline and month 15.

RESULTS: At month 15, the inotersen group experienced improvements from baseline in having severe impairments in numerous outcomes, including engaging in moderate activities, lifting/carrying groceries, climbing several flights of stairs, bending/kneeling/stooping, walking several hundred yards, reducing their amount of work and other activities, and health impacts on social activities (decreases ranging from 2% to 8% of the sample). The placebo group was more likely to experience severe impairments in these outcomes (increases ranging from 16% to 28% of the sample).

CONCLUSIONS: Among patients with hATTR amyloidosis, the group receiving inotersen for 15 months showed improvements in numerous concrete aspects of their daily lives, while the group receiving placebo showed worsening in these same activities.