Agminated Benign Vascular Tumor Successfully Treated with Trametinib
The novel treatment was administered at Sheba Medical Center after conventional therapies failed
Vascular tumors are caused by the neoplastic transformation of cells constituting vascular structures. Traditionally, treatment options include surgical intervention such as resection, embolization/sclerotherapy or laser therapy, and medical treatments such as corticosteroids, beta blockers and chemotherapy. However, the investigation of genetic mutations underlying vascular anomalies has recently provided new insights into the genetic etiology of these lesions, giving rise to personalized, targeted medical treatments that may potentially be safer and more effective.
A 28-year-old patient diagnosed with a reactive, disseminated vascular tumor originating from a small lesion under his eye was referred to Sheba Medical Center and failed to respond to conventional treatments. At this point, his treating physician, Dr. Shoshana Greenberger, Head of the Skin Disease Laboratory at Sheba Medical Center, opted to genetically sequence his tumor. The result indicated a somatic mutation in the NRAS gene.
With this result in mind, Dr. Greenberger requested that Trametinib, an oral MEK inhibitor normally reserved for patients with metastatic melanoma, be administered as compassionate treatment. “Trametinib is indicated for the treatment of other types of tumors, such as melanoma, and was yet to be tested on vascular tumors,” said Dr. Greenberger, who added: “Happily, the results were excellent.”
The treatment with Trametinib resulted in rapid regression of the tumor, and in less than 8 weeks it was completely gone. After the successful treatment, a case study was published in the British Journal of Dermatology.