Journal: Pediatric blood & cancer
This study assessed trametinib in five pediatric patients (median age 9) with complex lymphatic malformations, including kaposiform lymphangiomatosis and generalized lymphatic anomaly.
Treatment resulted in significant clinical improvement:
- Resolution of coagulopathy: platelet counts increased 54%-362%, D-dimer levels decreased >91%
- 80% had partial radiographic response
- Toxicity was minimal: one Grade 2 rash
NRAS mutations were found in 80% of cases, but clinical benefit was observed regardless of mutation status.
Trametinib appears to be an effective and safe first-line therapy for these conditions.