Patisiran improved functional capacity in patients with transthyretin cardiac amyloidosis


Administration of patisiran, a novel inhibitor of hepatic transthyretin synthesis, resulted in functional preservation in patients with cardiac transthyretin amyloidosis (ATTR). The patisiran group had better 6-minute walk test results and overall health status according to the KCCQ-OS questionnaire.

Researchers from Harvard Medical School, the Grossman School of Medicine, the Mayo Clinic, and the University of Copenhagen studied the effectiveness of the drug patisiran in patients with ATTR. Patisiran is an RNA interference drug that inhibits the synthesis of transthyretin by the liver. The study results were published in The New England Journal of Medicine.

After 12 months of treatment, the patisiran group had a smaller decrease in 6-minute walk distance: a difference of almost 15 m. KCCQ-OS scores improved in the patisiran group and worsened in the placebo group (average difference of 3.7 points). However, slightly more frequent infusion reactions, arthralgias, and muscle spasms were reported in the patisiran group.




Data from 360 patients with ATTR were analyzed. Participants were equally divided into two groups: receiving patisiran at a dose of 0.3 mg/kg body weight or placebo once every three weeks for 12 months. During the 1-year follow-up, changes in 6-minute walk distance and general health status based on the Kansas City Cardiomyopathy Questionnaire (KCCQ-OS), hospitalization rates, and all-cause mortality and mortality were assessed. mortality from cardiovascular complications.



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