Answers are grounded in this paper and cite their sources.
Treatment with trametinib improved clinical symptoms and normalized angiopoietin-2 in a child with KLA, suggesting its efficacy.
Kaposiform lymphangiomatosis (KLA) is a complex lymphatic anomaly with high morbidity and mortality. Angiopoietin-2 (Ang-2) levels are elevated in the blood of patients with KLA and can be used to aid in diagnosis and monitor treatment response. We report a male child with hydrocele, a left-sided pleural effusion, splenomegaly, and consumptive coagulopathy who was eventually diagnosed with KLA. Sirolimus did not provide a lasting therapeutic response, so treatment was switched to trametinib. The patient had an excellent response to trametinib with resolution of the pleural effusion, splenomegaly, and coagulopathy; normalization of Ang-2 levels; and significant improvement in clinical symptoms.
No takes yet. Share an insight, caveat, or question.
McDaniel et al. (2025) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: