Monitoring Arteriovenous Malformation Response to Genotype-Targeted Therapy.
Pediatrics
; 146(3)2020 09.
Article
in En
| MEDLINE
| ID: mdl-32859736
ABSTRACT
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.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Arteriovenous Malformations
/
Pyridones
/
Pyrimidinones
/
Spinal Cord Diseases
/
Proto-Oncogene Proteins p21(ras)
/
Protein Kinase Inhibitors
/
Genotype
Limits:
Adolescent
/
Humans
/
Male
Language:
En
Journal:
Pediatrics
Year:
2020
Document type:
Article