Decline in six-minute walk distance lower in patisiran versus placebo group at month 12<\/p>\n<\/h3>\n
By Elana Gotkine HealthDay Reporter<\/b><\/p>\n
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THURSDAY, Oct. 26, 2023 (HealthDay News) — For patients with transthyretin amyloidosis (ATTR amyloidosis), administration of an RNA interference therapeutic agent, patisiran, over 12 months results in preserved functional capacity, according to a study published in the Oct. 26 issue of the New England Journal of Medicine<\/em>.<\/p>\n Mathew S. Maurer, M.D., from the Columbia University Irving Medical Center in New York City, and colleagues conducted a phase 3 randomized trial involving patients with hereditary ATTR cardiac amyloidosis who were assigned to receive patisiran or placebo once every three weeks for 12 months (181 and 179 patients, respectively).<\/p>\n The researchers found that the decline in the six-minute walk distance was lower in the patisiran group than the placebo group at month 12 (Hodges-Lehmann estimate of median difference, 14.69 m); the Kansas City Cardiomyopathy Questionnaire-Overall Summary score increased and decreased in the patisiran and placebo groups, respectively (least-squares mean difference, 3.7 points). For the second secondary end point (composite of death from any cause, cardiovascular events, and change from baseline in the six-minute walk distance over 12 months), no significant benefits were observed. Compared with those receiving placebo, patients in the patisiran group more often had infusion-related reactions, arthralgia, and muscle spasms.<\/p>\n “Studies of longer duration are needed to determine whether the benefits observed with the reduction of transthyretin mediated by RNA interference are associated with a reduction in mortality and improved hospitalization outcomes in patients with ATTR cardiac amyloidosis,” the authors write.<\/p>\n The study was funded by Alnylam Pharmaceuticals, the manufacturer of patisiran.<\/p>\n Abstract\/Full Text (subscription or payment may be required)<\/a><\/p>\n Editorial (subscription or payment may be required)<\/a><\/p>\n <\/i><\/p>\n