Treatment satisfaction for gene silencing pharmacotherapies for the treatment of hereditary transthyretin amyloidosis with polyneuropathy

Background and Aims: Hereditary transthyretin amyloidosis (hATTR) is a rare, progressive, systemic, fatal condition in which misfolded transthyretin proteins form amyloid in tissues and organs, often manifesting in polyneuropathy (hATTR-PN). The FDA has approved 2 gene-silencing pharmacotherapies for hATTR-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 hATTR-PN (with and without accompanying cardiomyopathy) in the United States participated in an observational, online survey between January 1 and October 25, 2020. The Treatment Satisfaction Questionnaire for Medication, version II (TSQMvII), was administered to 29 patients currently being treated with inotersen (n=11) or patisiran (n=18). TSQMvII produces 4 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 (mean, 76.3 [SD=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]). Conclusions: While inotersen and patisiran were rated similarly in effectiveness and overall treatment satisfaction, inotersen was associated with less dissatisfaction with side effects and greater convenience, with the latter possibly reflecting differences in mode of administration (ie, at home vs visit to a clinical site), which may be particularly important during a pandemic.