e16252 Background: Hepatocellular carcinoma (HCC) with a solitary large tumor (≥ 5 cm) and microvascular invasion (MVI) is associated with a high risk of recurrence and poor survival prognosis. Currently, no effective adjuvant therapy has been established for this patient population. This study aimed to evaluate the efficacy and safety of adjuvant hepatic arterial infusion chemotherapy (HAIC) combined with donafenib in this high-risk group. Methods: This prospective, multicenter, single-arm, phase II trial was conducted at three hospitals in China. Patients with histologically confirmed treatment-naïve HCC were enrolled if they had a solitary tumor (≥ 5 cm) and MVI prior to surgical resection and had received no postoperative adjuvant therapies within 4-8 weeks before enrollment. Eligible patients were scheduled to receive adjuvant HAIC (FOLFOX4 regimen) combined with donafenib. The primary endpoint was the 2-year recurrence-free survival (RFS) rate. Secondary endpoints included RFS, overall survival (OS), and safety. Results: A total of 30 patients were enrolled. The median age was 59 years, and 93.3% of patients were male. Most patients were HBsAg-positive (73.3%), and 93.3% had Child-Pugh A liver function. The median maximum tumor diameter was 6.7 cm (IQR: 5.6-8.2), and 40.0% of patients had an AFP level > 400 ng/ml. After a median follow-up time of 22.4 months, tumor recurrence occurred in 9 patients, including 6 with intrahepatic recurrence and 3 with extrahepatic metastasis. The 1-, 2-, and 3-year RFS rates were 78.6%, 55.5%, and 55.5%, respectively, and the median RFS was not reached. The corresponding OS rates were 100.0%, 89.3%, and 79.4%, respectively, with the median OS not reached. In subgroup analysis, patients with AFP ≤ 400 ng/ml had numerically better RFS and OS than those with AFP > 400 ng/ml (1-, 2-, and 3-year RFS rates: 100.0%, 59.1%, and 59.1% vs. 54.5%, 45.5%, and 45.5%, respectively, p = 0.112; 1-, 2-, and 3-year OS rates: 100.0%, 100.0%, and 100.0% vs. 100.0%, 78.8%, and 65.6%, respectively, p = 0.110). The most common treatment-related adverse events (TRAEs) were hand-foot skin reaction (70.0%), diarrhea (43.3%), decreased platelet count (20.0%), hair loss (20.0%), and hypertension (16.7%). Grade 3 TRAEs occurred in 8 patients (26.7%), and no grade 4 or above TRAEs occurred. Conclusions: Adjuvant HAIC combined with donafenib showed encouraging efficacy and a well-tolerated safety profile in HCC patients with a solitary large tumor and MVI, suggesting a potential new adjuvant treatment strategy for this high-risk population. Clinical trial information: NCT04962958 .
Long et al. (Thu,) studied this question.