Abstract Details

Title RNAi Therapy in ATTRv Amyloidosis with Polyneuropathy (HELIOS-A): Patient-reported Experiences and Preferences

Topic General Neurology

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 4-001

Background Vutrisiran and patisiran are approved RNAi therapies for ATTRv amyloidosis with polyneuropathy. Vutrisiran is administered subcutaneously (Q3M) and patisiran intravenously (Q3W).

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.

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.

Authors/Disclosures
Maria Alejandra Gonzalez Duarte, MD, FAAN (NYU Dysautonomia Center) PRESENTER	Dr. Gonzalez Duarte has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Gonzalez Duarte has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Gonzalez Duarte has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Anylam . The institution of Dr. Gonzalez Duarte has received research support from Pfizer.
Laura Piera Obici (Fondazione IRCCS Policlinico San Matteo)	No disclosure on file
Lucia Galan Davila, MD	Dr. Galan Davila has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Galan Davila has received personal compensation in the range of $500-$4,999 for serving as a Consultant for pfizer. Dr. Galan Davila has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sobi. Dr. Galan Davila has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam. Dr. Galan Davila has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for pfizer. Dr. Galan Davila has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Akcea. Dr. Galan Davila has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for sobi. The institution of Dr. Galan Davila has received research support from Alnylam.
John L. Berk	John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca/IONIS. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eidos/BridgBio. John L. Berk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia Therapeutics. John L. Berk has received research support from Alnylam . John L. Berk has received research support from Ionis. John L. Berk has received research support from Eidos/Bridgbio.
Shaun Bender	No disclosure on file
Siddharth Jain	No disclosure on file
Varun Kumar (Alnylam Pharmaceuticals)	No disclosure on file
Kelley E. Capocelli, MD (Alexion Pharmaceuticals)	Dr. Capocelli has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Dr. Capocelli has stock in Alnylam Pharmaceuticals.
Michael J. Polydefkis, MD, FAAN (Johns Hopkins University School of Medicine)	Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceutical . Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen-Idec. Dr. Polydefkis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer.