Abstract Details

Title Open-label Study of Patisiran in Patients with hATTR Amyloidosis Post-Orthotopic Liver Transplant

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) Neuromuscular and Clinical Neurophysiology (EMG) Posters (7:00 AM-5:00 PM)

Poster/Presentation
Number 063

Objective To describe the efficacy, safety, and PK of patisiran in hATTR amyloidosis with polyneuropathy patients with disease progression post-OLT.

Background Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, life-threatening disease. Orthotopic liver transplant (OLT) suppresses mutant transthyretin (TTR) production to slow progression in early disease. Patisiran suppresses production of mutant and wild-type TTR and has been shown to halt or reverse polyneuropathy and improve quality of life in patients in APOLLO.

Design/Methods Phase 3b open-label study (NCT03862807) to evaluate the safety, efficacy, and pharmacokinetics (PK) of patisiran in patients with hATTR amyloidosis with polyneuropathy and disease progression post-OLT. Patients receive patisiran 0.3 mg/kg intravenously once every 3 weeks for 12 months. 6-month safety and efficacy analyses will be presented at the congress.

Results 23 patients were enrolled and received patisiran in the study. Median age was 58.0 years, 13 (56.5%) were males, and 15 (65.2%) had V30M mutation. At baseline, 1 (4.3%) patient had polyneuropathy disability (PND) score I, 9 (39.1%) had PND II, and 13 (56.5%) had PND IIIA/B. Five patients (21.7%) had New York Heart Association (NYHA) classification I, 5 (21.7%) had NYHA II, and none had NYHA III or IV at study baseline. The mean (SD) percent change in TTR at week 3 was -81.9% (11.3). Twenty-one (91.3%) patients experienced at least one adverse event (AE) and 3 (13.0%) patients experienced at least one serious AE. Five (21.7%) patients had AEs that were considered related to treatment. Updated 6-month efficacy and safety data will be presented at the congress.

Conclusions This study will continue to investigate the efficacy, safety, and PK of patisiran with the potential to address an unmet need in hATTR amyloidosis with polyneuropathy patients with disease progression post-OLT.

Authors/Disclosures
Seth Arum (Alnylam Pharmaceuticals) PRESENTER	Seth Arum has received personal compensation for serving as an employee of Alnylam Pharmaceuticals. Seth Arum has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wolters Kluwer.
Teresa Coelho, MD (Unidate Clinica de Paramoloidose Hospital)	Dr. Coelho has nothing to disclose.
	No disclosure on file
David D. Adams (APHP)	David D. Adams has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ALNYLAM. David D. Adams has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer.
	No disclosure on file
Anna Mazzeo, MD	Dr. Mazzeo has nothing to disclose.
	No disclosure on file
Violaine Plante-Bordeneuve, MD (CHU Henri Mondor)	Dr. Plante-Bordeneuve has nothing to disclose.
	No disclosure on file
	No disclosure on file
	No disclosure on file

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Abstract Details