RNAi Therapy in ATTRv Amyloidosis with Polyneuropathy (HELIOS-A): Patient-reported Experiences and Preferences
Alejandra Gonzalez-Duarte1, Laura Piera Obici2, Lucia Galan Davila3, John Berk4, Shaun Bender5, Siddharth Jain5, Varun Kumar5, Kelley Capocelli5, Michael Polydefkis6
1Department of Neurology, NYU Langone Health, 2Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, 3Department of Neurology, Hospital Universitario Clínico San Carlos, 4Boston University School of Medicine, 5Alnylam Pharmaceuticals, 6Department of Neurology, Johns Hopkins University School of Medicine
Objective:
We evaluated patient experiences and preferences about RNAi (RNA interference) treatment administration approaches for hereditary transthyretin-mediated (ATTRv; v for variant) amyloidosis with polyneuropathy.
Background:
Vutrisiran and patisiran are approved RNAi therapies for ATTRv amyloidosis with polyneuropathy. Vutrisiran is administered subcutaneously (Q3M) and patisiran intravenously (Q3W).
Design/Methods:
In HELIOS-A, patients with ATTRv amyloidosis with polyneuropathy were randomized to vutrisiran (Q3M) or patisiran (Q3W) for 18m with an extension phase where patients received vutrisiran (Q3M or Q6M) for 18m. A ‘Patient Experience Survey’ (PES) evaluating study treatment convenience in all patients at baseline/M9/M18 and a ‘Patient Preference Survey’ (PPS) was administered during the extension phase to evaluate preference between vutrisiran and patisiran.
Results:
113/122 vutrisiran-arm and 38/42 patisiran-arm patients completed the PES at M18; 95.6% and 99.1% of vutrisiran-arm PES respondents considered dosing frequency and time per dose administration, respectively, to be “somewhat”, “quite”, or “extremely” convenient, versus 71.1% and 57.9% of patisiran-arm respondents. PPS results at M9 were available in 27/37 patients who switched from patisiran to extension-phase vutrisiran treatment; 92.6% preferred vutrisiran over patisiran, with dose frequency (80.0%) and administration time (52.0%) being the most commonly cited reasons for preferring vutrisiran. The location of administration (i.e., infusion center, doctor’s office, home) was not a major factor driving vutrisian preference (8.0%).
Conclusions:
Majority of patients receiving vutrisiran or patisiran report favorable experiences regarding convenience, although vutrisiran is more commonly considered a convenient treatment option. The majority of patients switching from patisiran to vutrisiran prefer vutrisiran, primarily due to less frequent dosing; location of dose administration has limited impact on patient preference.