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Vandetanib therapy in medullary thyroid cancer.

机译:Vandetanib治疗髓质甲状腺癌。

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摘要

The U.S. Food and Drug Administration (FDA) approved vandetanib in April 2011 for the treatment of unresectable, locally advanced or metastatic medullary thyroid cancer (MTC). In Europe it was approved in March 2012, but only for the treatment of aggressive and symptomatic MTC. This small molecule is a tyrosine kinase inhibitor of several growth factors involved in cellular proliferation and angiogenesis, including the epidermal growth factor receptor (EGFR) and the vascular endothelial growth factor receptors 2 and 3 (VEGFR-2, VEGFR-3). In addition, vandetanib is an inhibitor of the RET (rearranged during transfection) gene, a proto-oncogene often mutated in familial MTC. Since MTC is a rare disease, for which no previous medical therapies are approved, vandetanib is the first drug shown to be effective in a large phase III trial treating patients with metastatic or locally advanced MTC. Common adverse events are diarrhea, nausea, hypertension, headache and QT prolongation that are manageable and are commonly outweighed by the benefits of vandetanib in terms of delaying disease progression and inducing tumor response.
机译:美国食品和药物管理局(FDA)于2011年4月批准了Vandetanib,用于治疗不可切除,局部晚期或转移性髓质甲状腺癌(MTC)。在欧洲,它于2012年3月批准,但仅用于治疗侵略性和症状的MTC。该小分子是涉及细胞增殖和血管生成的几种生长因子的酪氨酸激酶抑制剂,包括表皮生长因子受体(EGFR)和血管内皮生长因子受体2和3(VEGFR-2,VEGFR-3)。此外,Vandetanib是RET(转染期间重新排列)基因的抑制剂,通常在家族性MTC中经常突变的原癌基因。由于MTC是一种罕见的疾病,因此没有先前的医疗疗法被批准,Vandetanib是第一种在大型III期试验治疗转移或局部晚期MTC患者中有效的药物。常见的不良事件是腹泻,恶心,高血压,头痛和QT延长,可易于管理,并且通常在延迟疾病进展和诱导肿瘤反应方面的益处常见于Vandetanib的益处。

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