Hoy Sheridan M
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Target Oncol. 2024 May;19(3):473-480. doi: 10.1007/s11523-024-01059-8. Epub 2024 May 9.
Futibatinib (LYTGOBI) is an oral small molecule compound that selectively, irreversibly and potently inhibits the tyrosine kinase activity of fibroblast growth factor receptor (FGFR)1-4. It is approved in the EU, Japan and the USA for the treatment of adults with locally advanced or metastatic cholangiocarcinoma (CCA) harbouring an FGFR2 fusion or rearrangement who have progressed following systemic therapy. In the phase II part (FOENIX-CCA2) of a multinational phase I/II study in this patient population, monotherapy with futibatinib 20 mg once daily was associated with clinically meaningful and durable responses, sustained health-related quality of life (HR-QOL), and a manageable safety profile with supportive care and as-needed dose modifications. Indeed, hyperphosphataemia (the most common all grade and grade 3 treatment-related adverse event) was manageable with phosphate-lowering therapy and dose reductions or interruptions. Although further efficacy and tolerability data are expected, current evidence indicates that futibatinib is a valuable targeted therapy option for adults with locally advanced or metastatic CCA harbouring an FGFR2 fusion or rearrangement who have progressed following systemic therapy, a patient population with limited treatment options and poor life expectancy.
伏替巴替尼（LYTGOBI）是一种口服小分子化合物，可选择性、不可逆且强效地抑制成纤维细胞生长因子受体（FGFR）1 - 4的酪氨酸激酶活性。它在欧盟、日本和美国被批准用于治疗患有FGFR2融合或重排、经全身治疗后病情进展的局部晚期或转移性胆管癌（CCA）成人患者。在针对该患者群体的一项多国I/II期研究的II期部分（FOENIX - CCA2）中，每日一次口服20 mg伏替巴替尼单药治疗具有临床意义且持久的疗效、持续的健康相关生活质量（HR - QOL），以及通过支持治疗和必要的剂量调整可控制的安全性。事实上，高磷血症（最常见的所有级别和3级治疗相关不良事件）可通过降磷治疗以及剂量减少或中断来控制。尽管预计会有更多疗效和耐受性数据，但目前的证据表明，对于患有FGFR2融合或重排、经全身治疗后病情进展的局部晚期或转移性CCA成人患者，伏替巴替尼是一种有价值的靶向治疗选择，这是一个治疗选择有限且预期寿命较短的患者群体。
