A new study shows that the drug trametinib can significantly reduce mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by genetic mutations in the RAS/MAPK pathway.
American College of Cardiology released a study published in JACC: Basic to Translational Science on January 8, 2025. The study found that trametinib, a mitogen-activated protein kinase (MEK) inhibitor, reduced mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by pathogenic variants in the RAS/MAPK pathway.
This provides strong evidence for personalized treatment targeting the underlying genetic causes of RASopathies, a group of rare disorders that often lead to life-threatening cardiac complications. The study involved 61 children with severe RAS-HCM, comparing 30 children receiving trametinib to 31 receiving standard care. The results showed a significant reduction in death, cardiac transplantation, or the need for cardiac surgery in the trametinib group. No life-threatening adverse events were observed, although dermatologic and mucous membrane side effects were common but manageable
CARDIOLOGY GENETICS PEDIATRICS HYPERTROPHIC CARDIOMYOPATHY RASOPATHIES
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