Treatment Satisfaction For Gene Silencing Pharmacotherapies In Hereditary Transthyretin Amyloidosis With Polyneuropathy

Background and aims: Hereditary transthyretin amyloidosis (ATTRv) is a rare, progressive, systemic, fatal condition in which misfolded transthyretin proteins form amyloid in tissues and organs, often manifesting in polyneuropathy (ATTRv-PN). The FDA has approved two gene-silencing pharmacotherapies for ATTRv-PN: inotersen, administered subcutaneously;and patisiran, administered intravenously. Patient satisfaction for each treatment was examined during a period overlapping with the COVID-19 pandemic. Methods: Patients with ATTRv-PN (with and without accompanying cardiomyopathy) in the United States participated in an observational, online survey between January 1st and October 25th 2020. The Treatment Satisfaction Questionnaire for Medication, version II (TSQMvII) was administered to 29 patients currently treated with inotersen (n=11) or patisiran (n=19). TSQMvII produces four scale scores- Effectiveness, Side Effects, Convenience, and Global Satisfaction- ranging from 0 to 100. Higher scores indicate greater satisfaction. TSQMvII scores were descriptively compared between treatment groups. Results: Patients receiving inotersen indicated greater satisfaction with convenience than patients receiving patisiran (means 76.3 [standard deviation=19.4] vs. 58.6 [15.3], respectively), and less dissatisfaction with treatment side effects (86.1 [16.4] vs. 68.3 [19.0]). Ratings were comparable between treatments with respect to effectiveness (72.0 [21.5] vs. 67.1 [19.7]) and global satisfaction (78.0 [20.0] vs. 74.5 [21.7]). Conclusion: While inotersen and patisiran were rated similarly in effectiveness and overall treatment satisfaction, inotersen was associated with less dissatisfaction of side effects, and greater convenience, with the latter possibly reflecting differences in mode of administration (i.e., at home vs. visit to a clinical site), which may be particularly important during a pandemic.