Successful treatment of refractory chylothorax with MEK inhibitor trametinib in a child with Noonan syndrome: case report.
Ines HribernikTeresa BrooksAlix Dunlop-JonesJames R BenthamPublished in: European heart journal. Case reports (2023)
MEK inhibition with trametinib is emerging as a possible salvage treatment option for a subset of patients with Noonan syndrome and severe pulmonary lymphangiectasia. More experience is required to establish optimal treatment regimen and long-term outcomes.