Finch Elizabeth A, Elton Scott W, Huang Benjamin Y, Trembath Dimitri G, Blatt Julie
Departments of Pediatrics, Division of Pediatric Hematology-Oncology.
Neurosurgery, Division of Pediatric Neurosurgery.
J Pediatr Hematol Oncol. 2020 Mar;42(2):152-155. doi: 10.1097/MPH.0000000000001395.
Pleomorphic xanthoastrocytoma is a malignant brain tumor that has a good prognosis with complete resection but does not respond well to chemotherapy if there is residual tumor. BRAF V600E mutations are common in pleomorphic xanthoastrocytomas and provide an additional means for treatment when excision is not possible. Monotherapy with the BRAF V600E inhibitor vemurafenib has only been reported in a small number of cases and mostly in adults. We present the case of a 16-year-old male who responded to vemurafenib monotherapy initially and had an additional response to vemurafenib following progression after a brief time off the medication.
多形性黄色星形细胞瘤是一种恶性脑肿瘤，完整切除后预后良好，但如果有残留肿瘤，对化疗反应不佳。BRAF V600E突变在多形性黄色星形细胞瘤中很常见，当无法进行切除时，可为治疗提供额外手段。BRAF V600E抑制剂维莫非尼单药治疗仅在少数病例中报道过，且大多为成人病例。我们报告了一例16岁男性病例，该患者最初对维莫非尼单药治疗有反应，在停药一段时间后病情进展，再次使用维莫非尼仍有额外反应。
