High-intensity focused ultrasound alone or combined with transcatheter arterial chemoembolization for the treatment of hepatocellular carcinoma with unsuitable indications for hepatectomy and radiofrequency ablation: a phase II clinical trial

Objectives: This study aims to evaluate the efficacy and safety of high-intensity focused ultrasound (HIFU) alone or combined with transcatheter arterial chemoembolization (TACE) for patients with hepatocellular carcinoma (HCC) but were contraindicated for hepatectomy and radiofrequency ablation (RFA).

Methods: Patients between 20 and 80 years of age with 1-3 foci of HCC were selected. Included patients have had primary or recurrent liver lesions with no evidence of extra-hepatic metastasis prior to the study. Patients were treated with ultrasound-guided HIFU alone or HIFU combined with TACE (treated with TACE once within 4 weeks prior to receiving HIFU).

Results: Thirty-seven patients were enrolled, for a total of 45 lesions. The 2-year local control (LC) rate was 73.0% and the median LC time was 22 months. The 2-year progression-free survival (PFS) was 29.7% and the median PFS time was 9 months. Finally, the 2-year overall survival (OS) was 70.3%, and the median OS time was 24 months. The most common adverse events (AEs) were elevated liver enzymes, followed by fatigue, and pain, no grade 4 AEs or death occurred. Multivariate analysis showed that age, Child-Pugh class, and the number of tumors were independent prognostic factors for PFS and that the AFP levels and the number of tumors were significantly correlated with the OS.

Conclusions: This study indicates that the HIFU/HIFU combined with TACE treatment is safe, and is capable of achieving both a good LC rate and a considerably good prognosis. The procedure should be considered for patients who were deemed unsuitable for other local treatments.