CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
白血病患儿 CAR-T 细胞治疗后下呼吸道感染预测模型的构建
英文原题：Construction of a predictive model for lower respiratory tract infection in children with leukemia after chimeric antigen receptor T cell therapy.
查看英文原题
Construction of a predictive model for lower respiratory tract infection in children with leukemia after chimeric antigen receptor T cell therapy.
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研究概要
本研究构建了白血病患儿在接受 CAR-T 细胞治疗后 0-30 天内 LRTI 的风险预测模型。
中文摘要
**背景：**CAR-T（CAR-T）细胞治疗儿童复发/难治性白血病取得突破性进展。随着研究持续推进及临床应用增加，CAR-T 治疗后的感染逐渐受到关注。下呼吸道感染（LRTI）占全部感染事件的19.2%，并曾导致患者死亡。
本研究旨在探究接受CAR-T 治疗的白血病患儿发生LRTI的危险因素并建立风险预测模型。**方法：**回顾性收集2023年11月至2024年12月上海一家三级甲等儿童医院接受CAR-T 治疗的白血病患儿临床数据，分析LRTI独立危险因素并构建预测模型。采用Hosmer-Lemeshow检验和受试者工作特征（ROC）曲线下面积评估模型拟合度和区分度，并构建列线图以可视化模型。**结果：**研究纳入265例，CAR-T 治疗后0–30天内LRTI发生率为14.7%。LRTI危险因素包括血小板计数（PLT）<50×10^9/L（X1）、微小残留病（MRD）>20%（X2）、地塞米松剂量>10 mg（X3）及异体CAR-T（X4）。
回归方程为：LRTI发生风险y=1.027X1+1.079X2+1.187X3+1.096X4。Hosmer-Lemeshow检验χ²=2.674（P=0.95）。ROC曲线下面积为0.781（P<0.001），最大Youden指数为0.468，截断值0.139，敏感度76.9%，特异度69.9%。**结论：**本研究建立了白血病患儿CAR-T 治疗后0–30天内发生LRTI的风险预测模型，预测因素为PLT<50×10^9/L、MRD>20%、地塞米松用量>10 mg及异体CAR-T。
展开英文摘要原文
BACKGROUND
Chimeric antigen receptor T (CAR-T) cells have achieved breakthrough results in the treatment of refractory/relapsed leukemia in children. With the continuous development of research and increasing clinical application, infection events after CAR-T cell therapy have gradually attracted the attention of researchers. Lower respiratory tract infection (LRTI) events accounted for 19.2% of the total number of infection events and resulted in patient death. This study aims to investigate the risk factors of LRTI in children with leukemia who received CAR-T cell therapy and construct a risk predictive model.
METHODS
The clinical data of children with leukemia receiving CAR-T cell therapy in a tertiary A children's hospital in Shanghai from November 2023 to December 2024 were retrospectively collected, and the independent risk factors for LRTI were analyzed, and a risk predictive model was constructed. The Hosmer-Lemeshow test and the area under the receiver operating characteristic (ROC) curve were used to evaluate the fitting degree and discrimination of the predictive model, and a nomogram was constructed to visualize the model.
RESULTS
A total of 265 cases were included in this study, and the incidence of LRTI within 0-30 days after CAR-T cell therapy was 14.7%. The risk factors for developing LRTI were platelet count (PLT) <50 10 9 /L (X1), minimal residual disease (MRD) >20% (X2), dosage of dexamethasone greater than 10 mg (X3), and allogeneic CAR-T (X4), and the regression equation was: occurrence y (incidence of LRTI) = 1.027 X1 + 1.079 X2 + 1.187 X3 + 1.096 X4. Hosmer-Lemeshow test showed that 2 was 2.674 (P=0.95). The area under the ROC curve was 0.781 (P<0.001), the maximum Youden index was 0.468, the cut-off value was 0.139, the sensitivity was 76.9%, and the specificity was 69.9%.
CONCLUSIONS
In this study, a risk predictive model for LRTI in children with leukemia within 0-30 days after CAR-T cell therapy was constructed. The predictive factors were PLT <50 10 9 /L, MRD >20%, dexamethasone use greater than 10 mg, and allogeneic CAR-T.
论文信息
- 作者
- Tao L、He M、Zhang X、Sun J、Shen N、Shen B
- 第一作者单位
- Department of Hematology and Oncology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.China
- 通讯作者单位
- Department of Nursing, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.China
- 期刊
- Translational pediatrics2026 Jan 31
- 原文标识
- PubMed 41657452 · DOI 10.21037/tp-2025-503
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