Department of Medical Oncology, Hacettepe University Cancer Institute, Ankara, Turkey.
Department of Investigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center, Houston, USA.
Crit Rev Oncol Hematol. 2022 Jun;174:103700. doi: 10.1016/j.critrevonc.2022.103700. Epub 2022 May 6.
The immune checkpoint inhibitors (ICIs) entered treatment algorithms in most tumors. However, the data on the efficacy is limited in rare tumors with no phase III studies. We systemically reviewed the clinical trials evaluating the ICI efficacy in rare tumors and included a total of 47 clinical trials in this review. The ICIs demonstrated over 30% response rates in Merkel cell carcinoma and squamous cell carcinoma of the skin and became the standard of care. Additionally, the ICI efficacy was promising in thymic epithelial tumors and gestational trophoblastic neoplasia. In contrast, the ICI efficacy is limited in most sarcomas, germ cell tumors and low-grade neuroendocrine tumors. The ICI efficacy seemed to be improved with combinations targeting tumor microenvironment in sarcomas. The available evidence on ICI efficacy in rare tumors denote a need for better patient selection and novel combination strategies to improve outcomes.
免疫检查点抑制剂 (ICIs) 已进入大多数肿瘤的治疗方案。然而，在罕见肿瘤中，由于没有 III 期研究，关于其疗效的数据有限。我们系统地回顾了评估 ICI 在罕见肿瘤中的疗效的临床试验，本综述共纳入了 47 项临床试验。ICI 在 Merkel 细胞癌和皮肤鳞状细胞癌中显示出超过 30%的缓解率，成为标准治疗方法。此外，ICI 在胸腺瘤和妊娠滋养细胞肿瘤中也显示出良好的疗效。相比之下，ICI 在大多数肉瘤、生殖细胞肿瘤和低级别神经内分泌肿瘤中的疗效有限。ICI 联合靶向肿瘤微环境的疗效似乎有所改善。目前关于罕见肿瘤中 ICI 疗效的证据表明，需要更好的患者选择和新的联合策略来改善疗效。
