Regorafenib combined with a PD-1 inhibitor in the second-line setting for unresectable hepatocellular carcinoma in
Huaqiang Bi1, Jun Pei1, Kuansheng Ma1
1Department of Hepatobiliary Surgery, First Affiliated Hospital of Third Military Medical University (Army Medical University), Chongqing, China.
Background:
Most advanced hepatocellular carcinoma (HCC) cases administered molecular targeted agents and/or anti-programmed cell death-1 (PD-1) inhibitors have no response or develop resistance. Moreover, second-line therapies still cannot provide beneficial clinical outcomes. A pilot study assessing combined regorafenib and PD-1 inhibitor as second-line treatment of advanced HCC reported promising effectiveness.
Methods:
The current single-center, retrospective, real-world study was carried out between January 2019 and July 2021. Advanced HCC cases were administered second-line regorafenib combined with a PD-1 inhibitor or regorafenib alone were assessed. Progression-free survival (PFS), objective response rate (ORR), and disease control rate (DCR) were determined.
Results:
Totally 46 HCC cases were analyzed, most of whom underwent previous systemic treatment comprising targeted therapy and immunotherapy. Tumor response was evaluated in 25 and 21 individuals in the regorafenib + PD-1 inhibitor and regorafenib monotherapy groups, respectively: ORRs were 21.7% and 8.7%, and DCRs were 47.8% and 32.6%, respectively. Median PFS was markedly longer in the regorafenib plus PD-1 inhibitor group (11.5 months) compared with the regorafenib monotherapy group (5.1 months, P=0.049).
Conclusions:
This study suggested regorafenib and a PD-1 inhibitor in combination may provide significant clinical benefits in HCC cases showing progression following first-line treatment. Further analysis in real-world studies with large cohorts is warranted to confirm these findings.
Insights
Advanced hepatocellular carcinoma (HCC) patients showed improved outcomes with combined regorafenib and PD-1 inhibitors. This combination therapy offers better progression-free survival than regorafenib alone for refractory HCC.
Area of Science:
- Hepatobiliary Cancers
- Medical Oncology
- Immunotherapy
Background:
- Advanced hepatocellular carcinoma (HCC) often shows resistance to targeted agents and PD-1 inhibitors.
- Current second-line therapies for advanced HCC yield limited clinical benefits.
- A pilot study indicated promising effectiveness for combined regorafenib and PD-1 inhibitors in advanced HCC.
Purpose of the Study:
- To evaluate the efficacy of second-line regorafenib combined with a PD-1 inhibitor versus regorafenib monotherapy in advanced HCC.
- To compare progression-free survival (PFS), objective response rate (ORR), and disease control rate (DCR) between the two treatment groups.
Main Methods:
- A retrospective, real-world study conducted between January 2019 and July 2021.
- Analysis of 46 advanced HCC cases receiving either second-line regorafenib plus a PD-1 inhibitor or regorafenib alone.
- Assessment of tumor response, PFS, ORR, and DCR.
Main Results:
- The combination group (n=25) showed an ORR of 21.7% and DCR of 47.8%, compared to 8.7% ORR and 32.6% DCR in the monotherapy group (n=21).
- Median PFS was significantly longer in the combination group (11.5 months) versus the monotherapy group (5.1 months, P=0.049).
- Most patients had prior systemic treatment including targeted therapy and immunotherapy.
Conclusions:
- Combination therapy with regorafenib and a PD-1 inhibitor may offer significant clinical benefits for advanced HCC progressing after first-line treatment.
- Further large-scale, real-world studies are needed to validate these findings.
- This combination represents a potential improved second-line treatment strategy for advanced HCC.
