School of Pharmacy, University of Wisconsin, Madison, WI.
Division of Medical Oncology, Department of Internal Medicine, University of Kentucky, Lexington, KY.
Am J Health Syst Pharm. 2020 Feb 19;77(5):346-351. doi: 10.1093/ajhp/zxz329.
To provide an overview of fibroblast growth factor receptor (FGFR) gene alterations and the pharmacology, clinical effectiveness, dosage and administration, cost, and place in therapy of erdafitinib in bladder cancer.
Erdafitinib (Balversa, Janssen Pharmaceuticals) is a novel pan-FGFR inhibitor recently approved for the treatment of patients with advanced urothelial cancer with specific FGFR genetic alterations who have received at least one prior platinum-containing regimen. Erdafitinib binding to the FGFR2 and FGFR3 receptors inhibits FGF activity, resulting in cell death. Erdafitinib is available in tablet form, and the current recommended daily dosing is 8 mg, with dose escalation to 9 mg after 14 to 21 days of therapy if tolerated. A phase 2 clinical trial demonstrated that patients who received erdafitinib experienced on average 5.5 months of progression-free survival (95% confidence interval [CI], 4.2-6.0 months). In addition, 40% (95% CI, 31-50%) of patients responded to erdafitinib therapy. Patients receiving erdafitinib therapy should be monitored specifically for elevations in serum phosphate levels and changes in vision. Other adverse effects include anemia, thrombocytopenia, and electrolyte abnormalities.
Erdafitinib is the first small-molecule FGFR inhibitor approved for use in advanced bladder cancer.
概述成纤维细胞生长因子受体 (FGFR) 基因改变，以及厄达替尼在膀胱癌中的药理学、临床疗效、剂量和给药、成本及治疗地位。
厄达替尼（Balversa，杨森制药）是一种新型泛 FGFR 抑制剂，最近被批准用于治疗既往接受过至少一种含铂化疗方案治疗、存在特定 FGFR 基因改变的晚期尿路上皮癌患者。厄达替尼与 FGFR2 和 FGFR3 受体结合，抑制 FGF 活性，导致细胞死亡。厄达替尼为片剂，目前推荐的每日剂量为 8mg，如果耐受，治疗 14 至 21 天后可增加剂量至 9mg。一项 2 期临床试验表明，接受厄达替尼治疗的患者平均无进展生存期为 5.5 个月（95%置信区间[CI]：4.2-6.0 个月）。此外，40%（95%CI：31-50%）的患者对厄达替尼治疗有反应。接受厄达替尼治疗的患者应特别监测血清磷酸盐水平升高和视力变化。其他不良反应包括贫血、血小板减少和电解质异常。
厄达替尼是首个获批用于治疗晚期膀胱癌的小分子 FGFR 抑制剂。
