Trametinib normalizes angiopoietin-2 levels and successfully treats kaposiform lymphangiomatosis

曲美替尼可使血管生成素-2水平恢复正常,并成功治疗卡波西样淋巴管瘤病。

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Abstract

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.

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