Background/aim: The role of immune checkpoint inhibitors (ICIs; anti-PD1) in the treatment of childhood cancers is still evolving. The aim of this nationwide retrospective study was to assess the safety and effectiveness of ICIs used in a group of 42 patients, with a median age of 13.6 years, with various types of advanced malignancies treated in pediatric oncology centers in Poland between 2015 and 2023. Results: The indications for treatment with anti-PD1 were as follows: Hodgkin lymphoma (11); malignant skin melanoma (9); neuroblastoma (8); and other malignancies (14). At the end of follow-up, complete remission (CR) was observed in 37.7% (15/42) of children and disease stabilization in 9.5% (4/42), with a mean survival 3.6 (95% CI = 2.6–4.6) years. The best survival (OS = 1.0) was observed in the group of patients with Hodgkin lymphoma. For malignant melanoma of the skin, neuroblastoma, and other rare malignancies, the estimated 3-year OS values were, respectively, 0.78, 0.33, and 0.25 (p= 0.002). The best progression-free survival value (0.78) was observed in the group with malignant melanoma. Significantly better effects of immunotherapy were confirmed in patients ≥ 14 years of age and good overall performance ECOG status. Severe adverse events were observed in 30.9% (13/42) patients.
背景/目的：免疫检查点抑制剂（ICIs，特别是抗PD1药物）在儿童癌症治疗中的作用仍在不断探索中。本研究旨在通过一项全国性回顾性研究，评估2015年至2023年间波兰儿科肿瘤中心对42例中位年龄13.6岁、患有不同类型晚期恶性肿瘤的患儿使用ICIs的安全性和有效性。结果：抗PD1治疗的适应症分布如下：霍奇金淋巴瘤（11例）；恶性皮肤黑色素瘤（9例）；神经母细胞瘤（8例）；其他恶性肿瘤（14例）。随访结束时，37.7%（15/42）的患儿达到完全缓解，9.5%（4/42）实现疾病稳定，平均生存期为3.6年（95% CI = 2.6–4.6）。霍奇金淋巴瘤患儿组的总生存率最佳（OS = 1.0）。恶性皮肤黑色素瘤、神经母细胞瘤及其他罕见恶性肿瘤患儿的3年预估总生存率分别为0.78、0.33和0.25（p = 0.002）。恶性黑色素瘤组无进展生存率最佳（0.78）。研究证实，年龄≥14岁且ECOG全身状态评分良好的患儿接受免疫治疗效果显著更优。30.9%（13/42）的患儿出现严重不良事件。
肿瘤(癌症)患者之家