Multimodal Therapeutic Approach for a Severe Case of Kaposiform Lymphangiomatosis: From Procedural Interventions to Targeted Therapies

This case study describes a severe instance of kaposiform lymphangiomatosis (KLA) in a 10-year-old girl, highlighting the challenges and successes of a multimodal therapeutic approach. Initially treated with sirolimus, prednisone, and vincristine, the patient's condition worsened, necessitating a tracheostomy and frequent transfusions. A combination of endovascular embolizations and sclerotherapy stabilized her condition, but she remained steroid-dependent. Genetic testing revealed an NRAS mutation, leading to the introduction of the MEK inhibitor trametinib, which improved her condition significantly after 20 months of monotherapy. The study emphasizes the potential of trametinib as an alternative treatment for severe KLA, especially when traditional therapies like sirolimus and steroids are ineffective or cause long-term toxicities.