Hereditary transthyretin (ATTRv; v for variant) amyloidosis, also known as hATTR amyloidosis, is a rare, rapidly progressive, and fatal disease, in which continued progression of neuropathy and cardiomyopathy is associated with debilitating symptoms, impaired physical function, and decline in patients' quality of life (QOL).

This analysis from the phase 3 HELIOS-A study assessed the impact of treatment with the RNA interference therapeutic vutrisiran, which is approved for the treatment of the polyneuropathy of ATTRv amyloidosis, on measures of QOL and physical function in patients with ATTRv amyloidosis with polyneuropathy over 18 months.

Vutrisiran treatment significantly improved multiple measures of QOL and also demonstrated significant benefit in measures of gait speed, disability, performance status, and nutritional status, compared with the external placebo arm of the APOLLO study, which included a similar patient population, over 18 months.

The worsening from baseline in multiple measures of QOL and physical function observed in the external placebo group, together with the observation that patients with lower baseline polyneuropathy disability scores at the start of treatment with vutrisiran retained a better Norfolk Quality of Life-Diabetic Neuropathy score after 18 months, highlights that early and effective treatment of ATTRv amyloidosis with polyneuropathy is critical to minimize the progressive negative impact of the disease on QOL and physical function.

The findings of this study support the clinical benefit of vutrisiran as an effective treatment that can improve the lives of patients with ATTRv amyloidosis with polyneuropathy and emphasize the importance of early and effective treatment.