Abbreviations : CCLA, central conducting lymphatics anomaly; HVMC,
Hemangioma and Vascular Malformation Center at Cincinnati Children’s
Hospital Medical Center; GLA, generalized lymphatic anomaly; GSD,
Gorham-Stout Disease; IV, intravenously; KLA, kaposiform
lymphangiomatosis; LM, lymphatic malformation; PJP, Pneumocystis
jirovecii pneumonia; PO, per oral; SIADH, syndrome of inappropriate
antidiuretic hormone secretion; subQ, subcutaneously; VAC, Vascular
Anomalies Center at Texas Children’s Cancer and Hematology Centers.
†Common and notable lesser common complications listed. This is not an
extensive list of all potential side effects.
††Response to disease-modifying agents such as sirolimus and trametinib
for CCLA is unclear. EPHB4 mutation recently found in CCLA
suggests there may be a role for mTOR or MAPK/MEK inhibition.
*Dosing for infants and young children is different due to metabolism
differences. Recommended dosing for infants and young children under 2
years of age is based on pharmacokinetic studies by Mizuno et al.
[28]
**Suggested dosing based on clinical trials of trametinib use in
children with plexiform neurofibromas and gliomas.