JACC: Case Reports (Jul 2024)

Treatment of RAF1-Related Obstructive Hypertrophic Cardiomyopathy by MEK Inhibition Using Trametinib

  • Omid Kiamanesh, MD,
  • Steven C. Greenway, MD,
  • Franciscus Dicke, MD, MBA,
  • Brennan Ballantyne, MD,
  • Sasha Mitrovic, RDCS,
  • Kaitlin McGrath, MD,
  • James A. White, MD,
  • William D.T. Kent, MD,
  • Gregor Andelfinger, MD

Journal volume & issue
Vol. 29, no. 13
p. 102379

Abstract

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RASopathies cause nonsarcomeric hypertrophic cardiomyopathy via dysregulated signaling through RAS and upregulated mitogen-activated protein kinase activity. We provide the first report of the successful treatment of an adult with RAF1-associated hypertrophic cardiomyopathy using trametinib, a MEK inhibitor.

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