Surufatinib, a small-molecule inhibitor targeting VEGFR1, VEGFR2, VEGFR3, FGFR1, and CSF-1R, has exhibited satisfactory clinical effectiveness and favorable safety and tolerability profiles when utilized as monotherapy in the treatment of patients with biliary tract cancer (BTC) in the second-line setting.
This study included patients who have undergone resection for BTC with positive margins, as this patient population has a poor prognosis with no proper treatment recommendation. Zongli Zhang, MD, PhD, presented these preliminary results during ASCO GI. A real-world study assessed the efficacy and safety of surufatinib as a therapy for BTC.1,2
This is an ongoing single-arm, multicenter, open-label, real-world study (NCT05064852) conducted in China. It aims to enroll 200 patients with unresectable or margin-positive (MP) BTC. Physicians will administer surufatinib with or without combination as adjuvant (for patients with MP-BTC), first-line, or further-line therapy at 200 to 300 mg once daily in 28-day cycles.
The primary endpoint of this study is relapse-free survival (RFS) for patients with MP-BTC and progression-free survival (PFS) for those who had evaluable lesions. Secondary endpoints include objective response rate (ORR), disease control rate (DCR), median overall survival (OS), and safety. If available, tumor assessments will be performed every 2 cycles ± 7 days according to RECIST (version 1.1).
As of September 10, 2023, 56 patients had been enrolled, of which 28 had evaluable lesions. Patients have a median age of 64 years, and 40% are male. In comparison, 28 (50.0%), 24 (42.9%), and 4 (7.1%) patients received surufatinib as an adjuvant, first-line, and above-treatment, respectively.
This is an ongoing single-arm, multicenter, open-label, real-world study (NCT05064852) conducted in China. It aims to enroll 200 patients with unresectable or margin-positive (MP) BTC.
The location of primary lesions varied: 23 cases were intrahepatic cholangiocarcinoma (iCCA), 33 cases were extrahepatic cholangiocarcinoma (eCCA), and 33 cases were gallbladder cancer (GBC). The global median RFS/PFS was 10.4 (95% confidence interval [CI], 8.97-13.9) months.
The median PFS of patients who received surufatinib as adjuvant treatment was 11.3 (95% confidence interval [CI], 10.4-not achieved [NA]) months, and that of those who received surufatinib as a systematic treatment was 9.0 (95% CI, 7.8-10.8) months.
Patients with iCCA demonstrated a median PFS of 10.4 (5.8-NA) months, and those with eCCA or GBC demonstrated a median PFS of 10.4 (9.5-NA) months. The global median OS was 15.8 (12.4-NA) months.
Among those with primary lesions unresected (n=28), 3 (10.7%) patients achieved a complete response, 4 (14.3%) patients achieved a partial response, 16 (57.1%) patients had stable disease, and 5 (17.9%) patients suffered progressive disease. The ORR in patients receiving surufatinib as first-line therapy was 25%, and the DCR was 79.2%.
The ORR in patients receiving surufatinib in later lines of therapy was 25%, and the DCR was 100%. The ORR in patients receiving surufatinib in later lines of therapy was 25%, and the DCR was 100%.
The most common adverse events (AEs) included hypertension (8.9%) and anorexia (3.6%). There were no reported deaths from AEs, no severe AEs, and no patients discontinued treatment due to AEs. The AEs, however, are still being collected.
The real-world application of surufatinib revealed promising efficacy and manageable toxicity in patients suffering from BTC. Surufatinib may, therefore, be a beneficial therapy for patients with BTC.2
References
- Xu J, Bai Y, Sun H, et al. A single-arm, multicenter, open-label phase 2 trial of surufatinib in patients with unresectable or metastatic biliary tract cancer. Cancer. 2021;127(21):3975-3984.
- Zhang Z, Ma C, Li E, et al. Efficacy and safety of surufatinib in biliary tract cancer: preliminary results of a real-world study. San Francisco, CA, & online: presented at ASCO Gastrointestinal Cancers Symposium; January 18-20, 2024: poster C11.