To evaluate the safety and effectiveness of surgical closure of atrial septal defect in infants receiving trametinib for Noonan syndrome-associated hypertrophic cardiomyopathy.
Infants diagnosed with Noonan syndrome and hypertrophic cardiomyopathy were treated with trametinib.
Patients underwent surgical closure of the atrial septal defect after stabilization with trametinib.
Outcomes of surgical intervention were assessed post-operatively.
Surgical closure of the atrial septal defect was successful in the majority of cases.
Trametinib effectively stabilized infants for subsequent heart surgery.
Improved cardiac function was observed following the surgical procedure.
Abstract
Infants with severe presentations of NS-HCM and congenital heart defects can be medically stabilized with trametinib and then undergo successful structural heart surgery.