Why carry out this study?

Results from the phase 3 NEURO-TTR study found that 65 weeks of treatment with inotersen (Tegsedi™) led to improvements, relative to placebo, in the health-related quality of life (HRQOL) of patients with hereditary transthyretin amyloidosis associated with polyneuropathy (ATTRv-PN).

Because the long-term impact of inotersen on HRQOL for these patients has not yet been examined, the purpose of the current study was to evaluate longitudinal trends in HRQOL for patients in the NEURO-TTR trial who subsequently enrolled in an open-label extension (OLE) study for up to 2 years.

What was learned from the study?

The results of this interim analysis of data from the OLE study provide preliminary evidence that inotersen treatment stabilizes many aspects of HRQOL, including activities of daily living and physical, role, and social functioning, for up to 2–3 years, although due to limitations from the small sample size, further research within a larger patient sample is warranted.

Because previous studies observed no additional safety concerns or increased toxicity in patients exposed to inotersen for up to 5 years, inotersen may be a promising long-term treatment option for these patients.

While deterioration of most aspects of patients’ HRQOL was arrested by inotersen, it was not reversed, which highlights the importance of early diagnosis and treatment of this disease.