MEK Inhibition Beneficial for Children With Subset of Severe Hypertrophic Cardiomyopathy
By Elana Gotkine HealthDay Reporter
MONDAY, Jan. 13, 2025 -- For children with severe hypertrophic cardiomyopathy caused by gain-of-function RAS/mitogen-activated protein kinase (MAPK) mutations (RASopathy), trametinib reduces mortality and morbidity, according to a study published online Dec. 4 in JACC: Basic to Translational Science.
Cordula M. Wolf, M.D., from the German Heart Center Munich, and colleagues conducted a retrospective analysis of 61 patients younger than 18 years with RASopathy with severe hypertrophic cardiomyopathy. The patients received standard care plus MAPK inhibition (MEKi [trametinib], compassionate use; 30 patients) or standard care (31 patients).
The researchers observed a decrease in mortality and morbidity with improved cardiac status among those receiving trametinib versus standard-of-care treatment. The primary end point (composite of cardiac surgery for outflow tract resection, heart transplantation, or death) occurred in 17 and 87 percent of patients in the MEKi and control groups, respectively (hazard ratio, 0.09). Side effects were manageable and were not life-threatening.
"Taken together, our genotype-specific treatment results establish proof of principle to support the future development of treatment strategies for all RASopathy-associated cardiomyopathy, but potentially also for additional RASopathy manifestations," the authors write.
Several authors disclosed ties to biopharmaceutical companies, including Novartis, which manufactures trametinib.
Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.
© 2025 HealthDay. All rights reserved.
Posted January 2025
Read this next
CRC as First of Multiple Primary Malignancies Tied to Better Outcomes Than CRC as Only Malignancy
THURSDAY, June 26, 2025 -- Compared with colorectal cancer (CRC) presenting as an isolated primary or second primary malignancy, CRC presenting as the first of multiple primary...
Two-Hour Observation Probably Safe for Most Children With Anaphylaxis
THURSDAY, June 26, 2025 -- For most children who present to an emergency department with an acute allergic reaction requiring epinephrine, a two-hour observation period is...
CT Perfusion and CT Angiography Not Useful as Standalone Tests for Establishing Death by Neurologic Criteria
THURDAY, June 26, 2025 -- Neither computed tomography (CT) perfusion nor CT angiography meet the prespecified validation threshold as an ancillary test for death by neurologic...
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.