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供者来源 CAR-T 细胞疗法治疗异基因造血干细胞移植后儿童 B 细胞急性淋巴细胞白血病分子复发的长期生存与免疫重建

英文原题:Long-Term Survival and Immune Reconstitution of Donor-Derived Chimeric Antigen Receptor T-Cell Therapy for Childhood Molecular Relapse of B-Cell Acute Lymphoblastic Leukemia After Allogeneic Hematopoietic Stem Cell Transplantation.

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Long-Term Survival and Immune Reconstitution of Donor-Derived Chimeric Antigen Receptor T-Cell Therapy for Childhood Molecular Relapse of B-Cell Acute Lymphoblastic Leukemia After Allogeneic Hematopoietic Stem Cell Transplantation.

PubMed 2024/10/03(内容时间) Pediatr Hematol Oncol Q3 · IF 1.4(JCR 2025)

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中文摘要

异基因造血干细胞移植(allo-HSCT)后的可测量残留病(MRD)是急性淋巴细胞白血病(ALL)患者复发的独立危险因素。

本研究旨在评估 CAR-T 细胞治疗 allo-HSCT 后分子复发患者的疗效、安全性及免疫重建情况。研究纳入 11 例 allo-HSCT 后出现分子复发并接受 CAR-T 治疗的 B 细胞 ALL 患者。CAR-T 细胞输注 1 个月后,MRD 阴性率为 81.8%。CAR-T 治疗后桥接第二次 HSCT 的患者(n = 3),其 3 年无白血病生存率和 3 年总生存率呈较高趋势,优于未桥接移植者(n = 8;100% 对比 75.0%;95% 置信区间 45.0%–104.9%;p = 0.370)。未观察到治疗相关死亡。在未桥接第二次 HSCT、且截至末次随访仍处于完全缓解的患者中(n = 6),有 5 例未恢复正常免疫球蛋白浓度,中位随访时间为 43 个月。CAR-T 治疗可能是改善 allo-HSCT 后生存的一种安全有效策略;但对未桥接第二次 HSCT 的患者而言,长期低丙种球蛋白血症值得关注。

展开英文摘要原文

Measurable residual disease (MRD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an independent risk factor for relapse in patients with acute lymphoblastic leukemia (ALL).

This study aimed to assess the efficacy, safety, and immune reconstitution of chimeric antigen receptor T-cell (CAR-T) therapy in patients with molecular relapse after allo-HSCT. Eleven patients with molecular relapse of B-cell-ALL who underwent CAR-T therapy after allo-HSCT were enrolled. The rate of MRD negativity after a month of CAR-T infusion was 81. 8%. Patients who bridged to second-HSCT after CAR-T therapy ( n = 3) showed a trend of higher 3-year leukemia-free survival and 3-year overall survival than those who did not ( n = 8; 100% vs.

75. 0%; 95% CI, 45. 0-104. 9%; p = 0. 370). No treatment-related mortalities were observed. Among patients who did not bridge to second-HSCT and remained in complete remission until the last follow-up ( n = 6), five of them had not recovered normal immunoglobulin concentrations with a median follow-up of 43 months. CAR-T therapy may be a safe and effective treatment strategy to improve survival after allo-HSCT; however, the problem of prolonged hypogammaglobulinemia in patients who do not bridge to second-HSCT is worth noting.

论文信息

作者
Hu GH、Zuo YX、Suo P、Bai L、Zhang XH、Wang Y、Cheng YF、Huang XJ
单位
National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, Chinese Academic of Medical Sciences, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.China
期刊
Pediatric hematology and oncology2024 Nov
原文标识
PubMed 39360430 · DOI 10.1080/08880018.2024.2408535