免疫检查点抑制剂 (ICIs) 彻底改变了癌症治疗，并且与免疫相关不良事件 (irAE) 的关联已得到充分证实。然而，小脑 irAE 的定义不明确，其与副肿瘤性疾病的关系仍不清楚。我们的目标是 (i) 描述小脑 irAE 的特征； (ii) 将其与副肿瘤性小脑共济失调 (PCA) 进行比较。我们对 ICI 给药后出现新发、免疫介导、孤立/显性小脑功能障碍的患者进行了一项多中心、回顾性队列研究。此外，还按照 PRISMA 指南进行了系统审查。将小脑 irAE 病例与连续的 PCA 患者队列进行比较。总体而言，纳入了 35 名患者，其中 12 名是原始病例（男性：25/35 (71%)，中位年龄：65 [范围：20-82]）。最常见的肿瘤是非小细胞肺癌（12/35，34%）。 19/35 (54%) 采用了抗 PD1。 ICI 发病后平均 11 周出现症状。 15/31 名接受测试的患者 (48%) 检测到神经元抗体。 25/30 (83%) 的脑脊液呈炎症。磁共振成像显示 8/35 (23%) 的小脑高信号。 33/35例（94%）接受了免疫治疗，大多数患者的残余残疾得到改善（16/35，46%）。与一系列PCA（n = 15）相比，小脑irAE组与男性、肺癌（而不是妇科/乳腺癌）、孤立性共济失调和更好的结果显着更相关。我们提供了小脑 irAE 的详细特征。与 PCA 相比，在肿瘤关联、临床特征和结果方面存在差异。 ICI 组中的临床表现 - 抗体 - 肿瘤三联征仅部分反映了副肿瘤性疾病中描述的关联。© 2024。作者。

Immune checkpoint inhibitors (ICIs) have revolutionized cancer therapy, and the association with immune-related adverse events (irAEs) is well-established. However, cerebellar irAEs are poorly defined and their relationship with paraneoplastic disorders remains unclear. Our aim was (i) to characterize cerebellar irAE; (ii) to compare it with paraneoplastic cerebellar ataxia (PCA). We performed a multicenter, retrospective, cohort study of patients developing new-onset, immune-mediated, isolated/predominant cerebellar dysfunction after ICI administration. In addition, a systematic review following PRISMA guidelines was performed. Cerebellar irAE cases were compared with a consecutive cohort of patients with PCA. Overall, 35 patients were included, of whom 12 were original cases (males: 25/35 (71%), median age: 65 [range: 20-82]). The most frequent tumor was non-small cell lung cancer (12/35, 34%). Anti-PD1 were adopted in 19/35 (54%). Symptoms developed at a median of 11 weeks after ICI onset. Neuronal antibodies were detected in 15/31 patients tested (48%). Cerebrospinal fluid was inflammatory in 25/30 (83%). Magnetic resonance imaging showed cerebellar hyperintensities in 8/35 (23%). Immunotherapy was applied in 33/35 cases (94%), and most patients improved with residual disability (16/35, 46%). When compared with a series of PCA (n = 15), the cerebellar irAE group was significantly more associated with male sex, lung cancer (rather than gynecological/breast cancers), isolated ataxia, and a better outcome. We provide a detailed characterization of cerebellar irAE. Compared to PCA, differences exist in terms of tumor association, clinical features, and outcome. Clinical presentation-antibody-tumor triad in the ICI group only partially reflects the associations described in paraneoplastic disorders.© 2024. The Author(s).