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.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
司妥昔单抗治疗儿童、青少年和年轻成人急性淋巴细胞白血病 CAR-T 细胞治疗后的早期并发症
英文原题：Siltuximab for the treatment of early complications after chimeric antigen receptor T-cell therapy for acute lymphoblastic leukemia in children, adolescents, and young adults.
查看英文原题
Siltuximab for the treatment of early complications after chimeric antigen receptor T-cell therapy for acute lymphoblastic leukemia in children, adolescents, and young adults.
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研究概要
据我们所知，我们呈现了目前报道的规模最大的、因 B-ALL 接受 CAR-T 细胞疗法后发生 CRS 而使用 siltuximab 治疗的患者队列。
中文摘要
背景
细胞因子释放综合征（CRS）和免疫效应细胞相关神经毒性综合征（ICANS）是 CAR-T 细胞治疗相关并发症。Siltuximab 可直接结合白细胞介素 6（IL-6），作为 CRS 或 ICANS 一线治疗可能安全有效。
方法
回顾性研究纳入接受 CAR-T 治疗 B-ALL 后使用 siltuximab 的儿童、青少年及青年成人（AYA）患者。
结果
共纳入 118 例患者，其中 97 例发生 CRS（82%），26 例发生 ICANS（22%）。发生 CRS 的患者中，65 例（55%）接受治疗。65 例中 46 例（71%）接受抗 IL-6 药托珠单抗，19 例（29%）接受 siltuximab，用于治疗托珠单抗难治性 CRS（n = 13，68%）或作为 CRS 一线治疗（n = 6，32%）。接受 siltuximab 治疗者中有 9 例（47%）发生 ICANS。中位随访 12.1 个月时，7 例患者仍存活。
结论
据作者所知，这是报告中 CAR-T 治疗 B-ALL 后使用 siltuximab 治疗 CRS 的最大患者队列。siltuximab 的安全性特征及其抑制 IL-6 效应的作用提示，应在前瞻性临床试验中研究其作为一线治疗的应用。
展开英文摘要原文
BACKGROUND
Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) are complications associated with CAR T-cell therapy. Siltuximab directly binds interleukin-6 (IL-6) and may be safe and effective as first-line therapy for CRS or ICANS.
METHODS
A retrospective study was conducted on pediatric, adolescent and young adult (AYA) patients treated with siltuximab after CAR T-cell therapy for B-ALL.
RESULTS
A total of 118 patients treated were included: 97 patients developed CRS (82%), and 26 patients (22%) developed ICANS. Sixty-five of those that developed CRS (55%), received treatment. In 46/65 (71%), tocilizumab was administered as anti-IL-6 drug, and 19/65 (29%) patients received siltuximab to treat tocilizumab-refractory CRS (n = 13, 68%), or as first-line CRS treatment (n = 6, 32%). Nine patients treated with siltuximab (47%) developed ICANS. With a median follow-up of 12.1 months, 7 patients remained alive.
CONCLUSIONS
To the best of our knowledge, we present the largest reported cohort of patients treated with siltuximab for CRS following CAR T-cell therapy for B-ALL. Siltuximab's safety profile and its inhibition of IL-6 effects suggest that it should be investigated as first-line therapy in prospective clinical trials.
论文信息
- 作者
- Galán-Gómez V、González-Martínez B、Alonso-Saladrigues A、Rives S、Herrero B、Kwon M、Sánchez-Pina J、Minguillón J
- 第一作者单位
- Pediatric Hemato-Oncology Department, La Paz University Hospital, Paseo de La Castellana, 261, 28046, Madrid, Spain.Spain
- 通讯作者单位
- Pediatric Hemato-Oncology Department, La Paz University Hospital, Paseo de La Castellana, 261, 28046, Madrid, Spain. aperezmartinez@salud.madrid.org.Spain
- 期刊
- Experimental hematology & oncology2025 Apr 2
- 原文标识
- PubMed 40176077 · DOI 10.1186/s40164-025-00638-3
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