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
CD19 靶向 CAR-T 细胞治疗后的造血细胞移植用于儿童急性淋巴细胞白血病的缓解巩固或复发治疗
英文原题：Hematopoietic cell transplantation after CD19-directed CAR T-cell therapy for remission consolidation or relapse treatment in pediatric acute lymphoblastic leukemia.
查看英文原题
Hematopoietic cell transplantation after CD19-directed CAR T-cell therapy for remission consolidation or relapse treatment in pediatric acute lymphoblastic leukemia.
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中文摘要
B-ALL 在 CD19 靶向CAR-T 细胞治疗（CAR19）后复发仍是一项重大挑战。异基因造血细胞移植（HCT）既是预防 CAR19 后复发的手段，也是复发后的治疗手段。
然而，该人群中详细的 HCT 安全性和结局数据仍很匮乏。我们开展了一项回顾性研究，纳入 47 例因 CAR19 后缓解巩固治疗（抢先治疗队列，N=26）或复发治疗（复发队列，N=21）而接受首次 HCT 的 B-ALL 儿童及年轻成人患者。中位随访 4.1 年，抢先治疗队列和复发队列的 3 年无病生存率分别为 90% 和 64%。抢先治疗队列 3 年的总生存率、累积复发率和非复发死亡率分别为 95%、5% 和 5%，复发队列分别为 67%、20% 和 15%。3-4 级急性移植物抗宿主病（GvHD）的累积发生率在抢先治疗队列为 14%，在复发队列为 19%。在抢先治疗队列和复发队列中，100 天时存活患者分别有 24% 和 14% 发生慢性 GvHD。静脉闭塞病/肝窦阻塞综合征是最常见的非 GvHD 严重器官毒性，其累积发生率在抢先治疗队列为 10%，在复发队列为 31%。对于合适的患者，在 CAR19 后抢先应用 HCT，或将其作为 CAR19 后复发挽救治疗的一部分，均是实现 B-ALL 持久缓解的有效策略。
展开英文摘要原文
Relapse of B-cell acute lymphoblastic leukemia (B-ALL) after CD19-targeted chimeric antigen receptor T-cell therapy (CAR19) remains a substantial challenge. Allogeneic hematopoietic cell transplant (HCT) represents an approach for both post-CAR19 relapse prevention and relapse therapy.
However, there is a paucity of detailed HCT safety and outcome data in this population.
We conducted a retrospective review of 47 children and young adults with B-ALL who underwent first HCT for post-CAR19 remission consolidation (preemptive cohort, N=26) or relapse therapy (relapse cohort, N=21). With a median follow-up of 4. 1 years, 3-year disease-free survival was 90% in the preemptive cohort and 64% in the relapse cohort.
Overall survival, cumulative incidence of relapse, and non-relapse mortality at 3 years were 95%, 5%, and 5%, respectively, in the preemptive cohort and 67%, 20%, and 15%, repectively, in the relapse cohort. The cumulative incidence of grade 3-4 acute graft-versus-host disease (GvHD) was 14% in the preemptive cohort and 19% in the relapse cohort. Chronic GvHD developed in 24% and 14% of patients alive at 100 days in the preemptive and relapse cohorts, respectively.
Veno-occlusive disease/sinusoidal obstruction syndrome was the most common non-GvHD severe organ toxicity, with a cumulative incidence of 10% in the preemptive cohort and 31% in the relapse cohort. In appropriate patients, HCT can be an effective strategy for attaining durable B-ALL remission when used preemptively after CAR19 or as part of post-CAR19 relapse salvage therapy.
论文信息
- 作者
- Myers RM、Li Y、Liu H、Mumanachit S、Wang L、Leahy AB、Cain LE、DiNofia AM
- 第一作者单位
- Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA. myersrm@chop.edu.United States
- 通讯作者单位
- Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA. elgartenc@chop.edu.United States
- 期刊
- Haematologica2026 Sep 1
- 原文标识
- PubMed 41307125 · DOI 10.3324/haematol.2025.288303
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