Chen Wei-Yu, Chen Qing, Wang Chen-Chen, Zhang Chen-Yue, Chen Si-Kun, Meng Zhi-Qiang, Han Ping, Dong Shu, Chen Qi-Wen
Department of Integrative Oncology, Fudan University Shanghai Cancer Center Xiamen Hospital, Xiamen 361000, Fujian Province, China.
Department of Oncology, Shanghai Medical College, Shanghai 200032, China.
World J Hepatol. 2025 Aug 27;17(8):108533. doi: 10.4254/wjh.v17.i8.108533.
In randomized controlled trials (RCTs), the placebo arm has often been ignored as the attention tends to be focused on the treatment arm. We undertook a meta-analysis based on the data from the placebo arm in RCTs of hepatocellular carcinoma (HCC), the response rates and survival status, and adverse events (AEs) were summarized and evaluated.
To systematically evaluate the response rates, survival status and AEs in the placebo arms of RCTs for HCC.
A systematic search was performed on PubMed, Ovid MEDLINE, Embase and Cochrane Library to identify relevant trials evaluating the efficacy of drugs for the treatment of HCC, published until December 31, 2023. Statistical analysis was performed using R statistical software (version 4.3.2).
A total of 18 RCTs, involving 2390 patients, met the criteria for inclusion in the meta-analysis. The pooled overall disease control rate and objective response rate in the placebo group were 38% [95% confidence interval (CI): 33%-42%] and 1% (95%CI: 1%-2%), respectively. Overall survival and progression-free survival in the placebo group were 7.9 months (95%CI: 7.6-8.31 months) and 1.9 months (95%CI: 1.6-2.1 months), respectively. The incidence of grade 3 or 4 AEs was 37% (95%CI: 30%-43%). Additionally, the incidence of interruptions or dose reductions due to AEs was 20% (95%CI: 13%-27%), while the incidence of treatment discontinuation due to AEs was 9% (95%CI: 6%-12%).
Over one-third of advanced HCC patients exhibit therapy-free disease control, with placebo-arm AEs observed. These findings guide single-arm trials design and enhance patient acceptance of anticancer therapies.
在随机对照试验（RCT）中，安慰剂组常常被忽视，因为注意力往往集中在治疗组上。我们基于肝细胞癌（HCC）RCT中安慰剂组的数据进行了一项荟萃分析，总结并评估了缓解率、生存状况和不良事件（AE）。
系统评估HCC随机对照试验安慰剂组的缓解率、生存状况和不良事件。
在PubMed、Ovid MEDLINE、Embase和Cochrane图书馆进行系统检索，以识别评估药物治疗HCC疗效的相关试验，检索截至2023年12月31日发表的文献。使用R统计软件（版本4.3.2）进行统计分析。
共有18项RCT，涉及2390例患者，符合纳入荟萃分析的标准。安慰剂组的合并总体疾病控制率和客观缓解率分别为38%[95%置信区间（CI）：33%-42%]和1%（95%CI：1%-2%）。安慰剂组的总生存期和无进展生存期分别为7.9个月（95%CI：7.6-8.31个月）和1.9个月（95%CI：1.6-2.1个月）。3级或4级不良事件的发生率为37%（95%CI：30%-43%）。此外，因不良事件导致的中断或剂量减少的发生率为20%（95%CI：13%-27%），而因不良事件导致的治疗中断发生率为9%（95%CI：6%-12%）。
超过三分之一的晚期HCC患者表现出无需治疗的疾病控制，同时观察到安慰剂组的不良事件。这些发现为单臂试验设计提供了指导，并提高了患者对抗癌治疗的接受度。
