BackgroundImmune checkpoint inhibitors (ICI) have improved outcomes in a variety of adult cancers and are prescribed with increasing frequency across oncology. However, patterns of off‐label use of ICI in pediatrics remain unclear.MethodsThis is a single‐institution, retrospective cohort study evaluating off‐label ICI use in pediatric and young adult patients with cancer treated at our institution from 2014 to 2022. Response was based on clinician assessment derived from clinical records. Immune‐related adverse events (iRAEs) were classified according to CTCAE v5.0.ResultsWe identified 50 unique patients treated with off‐label ICI (28 with solid tumors, 20 with central nervous system (CNS) tumors, 2 with hematologic malignancies). At time of ICI initiation, only five patients (10%) had localized disease, and all but one patient was treated in the relapsed/refractory setting. All patients were treated with the FDA‐approved weight‐based dosing recommendations. Overall, there was disease control in 21 patients (42%), with best response including one complete response (melanoma), two partial responses (high‐grade glioma, CNS nongerminomatous germ cell tumor), and 18 patients with stable disease. Forty‐four patients (88%) eventually experienced disease progression. Among 22 patients (44%) experiencing iRAEs, 10 (20%) had a grade ≥3 irAE, 12 (24%) required corticosteroids, and 14 (28%) required ICI discontinuation. irAE occurrence was associated with significantly improved progression‐free survival (HR 0.35; 95% CI: 0.18 to 0.68; p = 0.002) and overall survival (HR 0.33; 95% CI: 0.17 to 0.66; p = 0.002).ConclusionsAt our institution, ICI was most commonly prescribed in the relapsed/refractory setting to patients with metastatic disease. The treatment was generally well‐tolerated in the pediatric population. The overall response rate was low, and the majority of patients eventually experienced disease progression. A few patients, however, had durable treatment responses. Further studies are needed to identify which pediatric patients are most likely to benefit from ICI.

中文翻译：

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在单一儿科癌症中心进行免疫检查点抑制剂治疗的超说明书处方

背景免疫检查点抑制剂（ICI）可以改善多种成人癌症的治疗效果，并且在肿瘤学领域的使用频率越来越高。然而，儿科超说明书使用 ICI 的模式仍不清楚。方法这是一项单机构回顾性队列研究，评估 2014 年至 2022 年在我们机构治疗的儿童和年轻癌症患者中超说明书 ICI 的使用。反应为基于临床医生从临床记录中得出的评估。免疫相关不良事件 (iRAE) 根据 CTCAE v5.0 进行分类。结果我们确定了 50 名接受超说明书 ICI 治疗的独特患者（28 名患有实体瘤，20 名患有中枢神经系统 (CNS) 肿瘤，2 名患有血液恶性肿瘤）。在 ICI 开始时，只有 5 名患者 (10%) 患有局部疾病，除一名患者外，所有患者均在复发/难治性环境中接受治疗。所有患者均按照 FDA 批准的基于体重的剂量建议进行治疗。总体而言，21 名患者 (42%) 获得了疾病控制，其中最佳缓解包括 1 名完全缓解（黑色素瘤）、2 名部分缓解（高级别胶质瘤、中枢神经系统非生殖细胞瘤），以及 18 名疾病稳定的患者。 44 名患者（88%）最终经历了疾病进展。在 22 名经历 iRAE 的患者 (44%) 中，10 名 (20%) 名 irAE ≥3 级，12 名 (24%) 需要皮质类固醇，14 名 (28%) 需要停止 ICI。 irAE 的发生与无进展生存期的显着改善相关（HR 0.35；95% CI：0.18 至 0.68；p= 0.002）和总生存率（HR 0.33；95% CI：0.17 至 0.66；p= 0.002）。结论在我们的机构，ICI 最常用于复发/难治性转移性疾病患者。该治疗在儿科人群中通常具有良好的耐受性。总体反应率较低，大多数患者最终出现疾病进展。然而，少数患者有持久的治疗反应。需要进一步研究来确定哪些儿科患者最有可能从 ICI 中受益。