Patisiran retains functional capacity in ATTR amyloidosis

THURSDAY, Oct. 26, 2023 (HealthDay News) — In patients with transthyretin amyloidosis (ATTR amyloidosis), the RNA interference therapeutic patisiran Functional ability can be retained for 26 periods beyond 12 months New England Journal of Medicine.

Mathew S. Maurer, MD, of Columbia University Irving Medical Center in New York City, and colleagues conducted a phase 3 randomized trial of patients with hereditary ATTR cardiac amyloidosis who were assigned to receive either patisiran or patisiran every three weeks. placebo for 12 months. 181 and 179 patients, respectively).

Researchers found that six-minute walk distance decreased less in the patisiran group than in the placebo group at 12 months (Hodges-Lehmann estimated median difference, 14.69 m); Kansas City Cardiomyopathy Questionnaire between patisiran and placebo groups Overall summary scores increased and decreased respectively (least squares mean difference, 3.7 points). For the second secondary endpoint (a composite of all-cause death, cardiovascular events, and change from baseline in six-minute walk distance over 12 months), no significant benefit was observed. Patients in the patisiran group experienced infusion-related reactions, arthralgia, and muscle spasms more frequently than patients who received placebo.

“Longer studies are needed to determine whether the benefits of RNA interference-mediated transthyretin reduction are associated with reduced mortality and improved hospital outcomes in patients with ATTR cardiac amyloidosis,” the authors wrote.

The study was funded by Alnylam Pharmaceuticals, the manufacturer of patisiran.

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