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
中国 B 细胞急性淋巴细胞白血病患者中 blinatumomab 治疗模式、疗效和安全性的真实世界证据
英文原题：Real-world evidence on treatment pattern, effectiveness, and safety of blinatumomab in Chinese patients with B-cell acute lymphoblastic leukemia.
查看英文原题
Real-world evidence on treatment pattern, effectiveness, and safety of blinatumomab in Chinese patients with B-cell acute lymphoblastic leukemia.
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中文摘要
Blinatumomab 对 B 细胞急性淋巴细胞白血病（B-ALL）有效，但相关真实世界数据有限。本回顾性研究提供了 blinatumomab 在中国新诊断（ND）及复发/难治性（R/R）B-ALL 患者中的治疗模式、有效性和安全性真实世界数据。纳入 2021 年 8 月至 2023 年 6 月期间在初治或 R/R 阶段至少接受一剂 blinatumomab 的 B-ALL 患者。主要结局为 blinatumomab 治疗模式；关键次要结局包括完全缓解（CR）/血细胞恢复不完全的 CR（CRi）率、微小残留病（MRD）阴性率、中位无事件生存期（EFS）和安全性。研究共纳入 96 例 B-ALL 患者：53 例（55.2%）为 ND 组，43 例（44.8%）为 R/R 组。
中位治疗时长为 1 个周期（范围 1–5）。多数患者在 blinatumomab 后接受化疗、异基因 HSCT 或试验性 CAR-T。ND 患者采用 blinatumomab 诱导治疗后 CR/CRi 率为 100%；87.2% 在两个周期内达到 MRD 阴性。R/R 再诱导患者 CR/CRi 率为 50%，MRD 阴性率为 64.2%；R/R 巩固治疗患者 MRD 阴性率为 90.9%。ND 和 R/R 患者的中位 EFS 均尚未达到；1 年 EFS 率分别为 90.8%（95% CI 67%–97%）和 55.1%（95% CI 30%–74%）。12.5% 患者观察到 3 级不良事件。真实世界中 blinatumomab 疗效良好且安全性可耐受。
展开英文摘要原文
Blinatumomab is efficacious in patients with B-cell acute lymphoblastic leukemia (B-ALL), yet limited real-world data exists in this context. This retrospective study provided real-world data on the treatment pattern, effectiveness, and safety of blinatumomab in Chinese patients with newly diagnosed (ND) and relapsed/refractory (R/R) B-ALL. Patients with B-ALL who received at least one dose of blinatumomab in frontline or R/R settings between August 2021 and June 2023 were included. The primary outcome was the treatment pattern of blinatumomab. Key secondary outcomes included complete remission (CR)/CR with incomplete blood cell recovery (CRi) rate, minimal residual disease (MRD) negativity, median event-free survival (EFS), and safety. The study included 96 patients with B-ALL; 53 (55. 2%) patients were in the ND group and 43 (44.
8%) patients were in the R/R group. The median treatment duration was one cycle (range: 1-5). Most patients underwent chemotherapies, allo-HSCT, or experimental CAR-T following blinatumomab. The ND patients using blinatumomab induction therapy achieved 100% CR/CRi rate; 87. 2% achieved MRD negativity within two cycles of blinatumomab. In R/R re-induction patients, the CR/CRi rate was 50%; MRD negativity rate was 64.
2%. In R/R patients using blinatumomab for consolidation, MRD negativity rate was 90. 9%. The median EFS was not reached in both ND and R/R patients; 1-year EFS rate was 90. 8% (95% CI: 67%, 97%) and 55. 1% (95% CI: 30%, 74%), respectively. Grade 3 adverse events were observed in 12. 5% patients. Blinatumomab was found to be effective with a tolerable safety profile in real world setting.
论文信息
- 作者
- Zhou H、Wu X、Yang Z、Lu S、Zhang X、Yang X、Chen S、Wu D
- 第一作者单位
- Department of Hematology, The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, National Clinical Research Center of Hematologic Diseases, No 188, Shizi Street, Suzhou, Jiangsu, 215006, China.China
- 通讯作者单位
- Department of Hematology, The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, National Clinical Research Center of Hematologic Diseases, No 188, Shizi Street, Suzhou, Jiangsu, 215006, China. mm85124@sina.com.China
- 期刊
- Investigational new drugs2024 Jun
- 原文标识
- PubMed 38662275 · DOI 10.1007/s10637-024-01435-1