Newly Developed Drugs for Hepatocellular Carcinoma Expanded the Use of Systemic Therapy: An Interrupted Time Series Analysis Using an Electronic Medical Record in Japan

日本基于电子病历的间断时间序列分析:新型肝细胞癌药物扩大了全身治疗的应用范围

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Abstract

Introduction: Systemic therapy options for hepatocellular carcinoma (HCC) have rapidly expanded, transforming the treatment landscape. However, real-world changes in treatment choices remain unclear. This study aimed to determine the current treatment choices for HCC in Japan, considering age and liver functional reserves. METHODS: We conducted an interrupted time series analysis using electronic medical records in Japan to assess the overall changes in the proportions of systemic therapy among the initial treatments for HCC following the approval of lenvatinib in 2018 and atezolizumab plus bevacizumab (atezo/bev) in 2020, stratified by age and modified albumin-bilirubin (mALBI) grade. This study included patients who were diagnosed with HCC between 2015 and 2022. We also assessed 2-year survival rates. RESULTS: In the interrupted time series analysis, the proportions of systemic therapy among the initial treatments for HCC increased by 3.96% (95% confidence interval: 2.75, 5.17, p < 0.001) following lenvatinib approval, with a 1.00% (0.45, 1.55, p = 0.002) slope change per 6 months. There was a 4.27% (1.69, 6.86, p = 0.004) increase in systemic therapy use following atezo/bev approval and a 0.27% (-0.66, 1.20, p = 0.53) slope change. In age subgroups with a cutoff of 75 years, the largest increase in systemic therapy use was 6.94% (4.46, 9.42) in the elderly group following atezo/bev approval (p = 0.189). Increases in systemic therapy use following lenvatinib approval in patients with mALBI grades 2b or 3 and 1 or 2 were 10.0% (6.70, 13.42) and 0.80% (-1.16, 2.77), respectively (p < 0.001). There was no apparent change in the overall 2-year survival or in any subgroup before and after approval. CONCLUSION: Newly developed drugs for HCC would expand the population for systemic therapy. The optimal population for systemic therapy should be explored based on long-term survival considering age and liver functional reserve heterogeneity.

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