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Effective Use of ALK Inhibitors in EML4::ALK-Positive Lymphatic Malformations
Beth Apsel Winger1,2, Christopher F Dowd3, Kristin A Shimano2
1Department of Clinical Pharmacy, UCSF, San Francisco, California, USA.
Pediatric Blood & Cancer
|November 12, 2024
Abstract:
Genetically targeted medications are emerging as important therapies for lymphatic malformations (LMs) unresponsive to sirolimus. We describe two patients with EML4::ALK-positive LMs, one with Gorham Stout disease and one with a large genitourinary (GU) LM, who were successfully treated with ALK inhibitors. This report adds ALK inhibitors to the growing toolbox of molecularly targeted therapies for LMs.

