Immune checkpoint inhibitors (ICIs) have revolutionized the treatment of different malignancies. However, their efficacy in advanced adrenocortical carcinoma (ACC) remains uncertain. Thus, we conducted a systematic review and meta-analysis to summarize the efficacy and tolerability of ICIs in patients with advanced ACC. We searched PubMed, Scopus, and CENTRAL for studies that used ICIs in ACC. Studies with more than five patients were included in the meta-analysis of the objective response rate (ORR), disease control rate (DCR), overall survival (OS), progression-free survival (PFS), and grade 3/4 adverse events. Twenty studies with 23 treatment arms and 250 patients were included. Single-agent anti-PD1 or anti-PD-L1 treatment was utilized in 13 treatment arms, whereas an anti-PD1 or anti-PD-L1 and anti-CTLA4 combination was used in 4 treatment arms. Other anti-PD1- or anti-PD-L1-based combinations were used in five treatment arms. The ORR was 14% (95% CI = 10–19%, I2= 0%), and the DCR was 43% (95% CI = 37–50%, I2= 13%). The combination anti-PD1- or anti-PD-L1-based treatment strategies did not correlate with higher responses compared with monotherapy. The median OS was 13.9 months (95% CI = 7.85–23.05), and the median PFS was 2.8 months (95% CI = 1.8–5.4). ICIs have a modest efficacy in advanced ACC but a good OS. Further studies are needed to investigate predictive biomarkers for ICI response and to compare ICI-based strategies with the current standard of care.
免疫检查点抑制剂（ICIs）已彻底改变了多种恶性肿瘤的治疗格局，但其在晚期肾上腺皮质癌（ACC）中的疗效仍不明确。为此，我们开展了一项系统综述与荟萃分析，旨在总结ICIs在晚期ACC患者中的疗效与耐受性。我们检索了PubMed、Scopus及CENTRAL数据库中关于ACC应用ICIs的研究。纳入患者数超过5例的研究被用于客观缓解率（ORR）、疾病控制率（DCR）、总生存期（OS）、无进展生存期（PFS）及3/4级不良事件的荟萃分析。 最终共纳入20项研究，涵盖23个治疗队列，合计250例患者。其中13个队列采用单药抗PD1或抗PD-L1治疗，4个队列采用抗PD1/PD-L1联合抗CTLA4治疗，另有5个队列采用其他以抗PD1或抗PD-L1为基础的联合治疗方案。汇总分析显示，ORR为14%（95% CI = 10–19%，I²= 0%），DCR为43%（95% CI = 37–50%，I²= 13%）。与单药治疗相比，基于抗PD1或抗PD-L1的联合治疗方案并未显著提升疗效。中位OS为13.9个月（95% CI = 7.85–23.05），中位PFS为2.8个月（95% CI = 1.8–5.4）。 研究表明，ICIs在晚期ACC中疗效有限，但总生存期表现良好。未来需进一步探索预测ICI疗效的生物标志物，并将基于ICI的治疗策略与现行标准治疗方案进行比较研究。
肿瘤(癌症)患者之家