Journal
PEDIATRICS
Volume 146, Issue 3, Pages -Publisher
AMER ACAD PEDIATRICS
DOI: 10.1542/peds.2019-3206
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We use a novel imaging biomarker to monitor treatment response to genotype-targeted therapy of a KRAS mutant metameric AVM with an MEK inhibitor. Arteriovenous malformations (AVMs) have recently been reported to have a high incidence of somatic KRAS mutations suggesting potential for treatment with mitogen-activated protein kinase inhibitors. In this case report, we describe genotype-targeted treatment of a KRAS mutant metameric AVM in a patient with Cobb syndrome using the mitogen-activated protein kinase inhibitor trametinib. Therapeutic response was monitored with phase-contrast magnetic resonance angiography to quantify AVM arterial inflow as an imaging biomarker. Treatment with trametinib resulted in a substantial decrease in blood flow to the AVM, with a >75% reduction in arterial inflow after 6 months of trametinib therapy.
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