High-grade astrocytomas are malignant and aggressive, with limited treatment options. Treatment is geared not only toward increasing patient’s overall survival but also in delaying or preventing neurological disability, a cause of significant morbidity. Increasingly, targeted and customized treatment approaches, especially for recurrent disease, are being explored. Here we present a successful outcome in a young patient with rapidly progressive disease who responded to targeted treatment based on genetic sequencing and circulating tumor DNA markers, given the inaccessibility of the tissue to biopsy. Molecular testing on tissue, serum or CSF may be helpful in identifying unique targets in these complex patients.

中文翻译：

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复发性间变性星形细胞瘤的分子靶向治疗——病例报告

高级别星形细胞瘤是恶性和侵袭性的，治疗选择有限。治疗不仅旨在提高患者的总体生存率，还旨在延迟或预防神经功能障碍，这是导致严重发病的原因。越来越多的有针对性和定制化的治疗方法，特别是针对复发性疾病，正在被探索。在这里，鉴于组织无法进行活检，我们在一名患有快速进展性疾病的年轻患者中展示了成功的结果，该患者对基于基因测序和循环肿瘤 DNA 标记的靶向治疗做出了反应。对组织、血清或脑脊液进行分子检测可能有助于确定这些复杂患者的独特靶点。