Department of Neurology, Henry Ford Hospital, Detroit, MI, USA.
J Neurooncol. 2011 Jan;101(2):307-10. doi: 10.1007/s11060-010-0244-3. Epub 2010 Jun 4.
There is a need for effective systemic therapy for central nervous system (CNS) hemangioblastomas (HBs). We report a case of erlotinib therapy for CNS HBs in a patient with von Hippel-Lindau disease, in whom the HBs were associated with diffuse leptomeningeal seeding. We provide the first report of paired serum and cerebrospinal fluid (CSF) levels of erlotinib while on standard dosing. The patient exhibited neurologic and imaging signs of recurrent CNS HBs and progressive leptomeningeal metastasis following surgery, radiation, and stereotactic radiosurgery. The patient was treated with erlotinib 150 mg daily. The patient achieved a minor response to erlotinib therapy, including clinical improvement, reduction in size of two enhancing brain lesions (one of which, however, proved at autopsy to be radiation necrosis) and stabilization of leptomeningeal enhancement. In addition, the CSF white count improved. The duration of response was 9 months. The median plasma and CSF levels of erlotinib while on treatment were 1146.06 and 247.83 ng/ml, respectively (CSF 21.6% of plasma). Erlotinib may have antitumor activity in CNS HBs.
中枢神经系统（CNS）血管母细胞瘤（HBs）需要有效的全身治疗。我们报告了一例贝伐单抗治疗von Hippel-Lindau 病相关弥漫性软脑膜播散性中枢神经系统 HBs 的病例，患者同时存在 CFS 水平的配对血清和脑脊液（CSF）水平。该患者在标准剂量下表现出复发性 CNS HBs 和进行性软脑膜转移的神经和影像学征象，手术、放疗和立体定向放疗后。该患者接受了每日 150mg 的厄洛替尼治疗。患者对厄洛替尼治疗有轻微反应，包括临床改善、两个增强脑病变的大小缩小（其中一个经尸检证实为放射性坏死）和软脑膜增强稳定。此外，CSF 白细胞计数有所改善。反应持续时间为 9 个月。治疗期间厄洛替尼的中位血浆和 CSF 水平分别为 1146.06 和 247.83ng/ml（CSF 为血浆的 21.6%）。厄洛替尼可能对中枢神经系统 HBs 具有抗肿瘤活性。
