Dana-Farber Cancer Institute, Center for Salivary and Rare Head and Neck Cancers, Boston, Massachusetts.
Samsung Medical Center, Department of Hematology and Oncology, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Clin Cancer Res. 2023 Nov 14;29(22):4555-4563. doi: 10.1158/1078-0432.CCR-23-1030.
This open-label, single-arm, phase II study evaluated the vascular endothelial growth factor receptor 2 (VEGFR2) tyrosine kinase inhibitor (TKI) rivoceranib in patients with recurrent or metastatic (R/M) adenoid cystic carcinoma (ACC).
Eligible patients had confirmed disease progression per Response Evaluation Criteria in Solid Tumors (RECIST) with ≥20% increase in radiologically or clinically measurable lesions or appearance of new lesions within the preceding 6 months. Patients received oral rivoceranib 700 mg once daily. Primary outcomes were objective response rate (ORR) by investigator review and by blinded independent review committee (BIRC).
Eighty patients were enrolled and 72 were efficacy evaluable. Seventy-four patients had distant metastases and 49 received prior systemic treatment (14 received VEGFR TKIs). Per investigator and BIRC, respectively, ORR was 15.3% [95% confidence interval (95% CI), 7.9-25.7] and 9.7% (95% CI, 4.0-19.0); median duration of response was 14.9 months (95% CI, 4.9-17.3) and 7.2 months (95% CI, 3.5-8.4); and median progression-free survival was 9.0 months (95% CI, 7.3-11.5) and 9.0 months (95% CI, 7.7-11.5). Grade ≥3 treatment-related adverse events occurred in 56 patients (70.0%); the most common were hypertension (34, 42.5%) and stomatitis (6, 7.5%). Four grade 5 events occurred with one attributed to rivoceranib (epistaxis). Sixty-eight patients (85.0%) had ≥1 dose modifications and 16 patients (20.0%) discontinued rivoceranib for toxicity.
In patients with progressing R/M ACC, rivoceranib demonstrated antitumor activity and a manageable safety profile consistent with other VEGFR TKIs.
本开放标签、单臂、二期研究评估了血管内皮生长因子受体 2（VEGFR2）酪氨酸激酶抑制剂（TKI）rivoceranib 在复发或转移性（R/M）腺样囊性癌（ACC）患者中的疗效。
符合条件的患者根据实体瘤反应评价标准（RECIST）确认疾病进展，在过去 6 个月内影像学或临床可测量病变增加≥20%或出现新病变。患者接受口服 rivoceranib 700mg，每日一次。主要终点为研究者评估和盲法独立审查委员会（BIRC）评估的客观缓解率（ORR）。
共纳入 80 例患者，72 例可进行疗效评估。74 例患者有远处转移，49 例患者接受过系统治疗（14 例接受过 VEGFR TKI 治疗）。根据研究者和 BIRC 的评估，ORR 分别为 15.3%（95%CI，7.9-25.7）和 9.7%（95%CI，4.0-19.0）；中位缓解持续时间分别为 14.9 个月（95%CI，4.9-17.3）和 7.2 个月（95%CI，3.5-8.4）；中位无进展生存期分别为 9.0 个月（95%CI，7.3-11.5）和 9.0 个月（95%CI，7.7-11.5）。56 例（70.0%）患者发生≥3 级与治疗相关的不良事件；最常见的是高血压（34 例，42.5%）和口腔炎（6 例，7.5%）。4 例 5 级事件，其中 1 例归因于 rivoceranib（鼻出血）。68 例（85.0%）患者有≥1 次剂量调整，16 例（20.0%）因毒性而停止使用 rivoceranib。
在进展性 R/M ACC 患者中，rivoceranib 显示出抗肿瘤活性和可管理的安全性，与其他 VEGFR TKI 一致。
