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
CAR-T 治疗后序贯造血干细胞移植可改善复发/难治性费城染色体阳性 B 细胞急性淋巴细胞白血病患儿的生存
英文原题：CAR-T Therapy Followed by Hematopoietic Stem Cell Transplantation Can Improve Survival in Children Relapsed/Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia.
查看英文原题
CAR-T Therapy Followed by Hematopoietic Stem Cell Transplantation Can Improve Survival in Children Relapsed/Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia.
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研究概要
对于 Ph 阳性 B-ALL 儿童患者，CAR-T 治疗后序贯 allo-HSCT 是一种安全有效的治疗。
中文摘要
背景
费城染色体（Ph）阳性 B 细胞急性淋巴细胞白血病（ALL）完全缓解（CR）率较高，但复发和可测量残留病持续存在仍是严重问题。本研究评估儿童 Ph 阳性 ALL 患者接受 CD19 特异性嵌合抗原受体（CAR-19）T 细胞治疗后再接受造血干细胞移植（HSCT）的 CR 率、可测量残留病阴性率、治疗结局和安全性。
方法
研究于 2013 年 9 月至 2021 年 1 月在北京大学人民医院开展，纳入 13 例接受 CAR-T 后续异基因 HSCT 的复发/难治性 Ph 阳性 B-ALL 患者，重点评估总生存期和 CR。
结果
CAR-T 治疗与异基因 HSCT 的中位间隔为 58 天。CAR-T 输注后 1 个月，患者 CR 率为 100%，流式细胞术阴性率为 84.62%，BCR-ABL 阴性率为 53.85%。所有患者在 HSCT 后 6 个月均达到主要分子学缓解。中位随访 45 个月后，3 年总生存率（OS）为 66.7%，3 年无病生存率（DFS）为 61.5%。移植前 BCR-ABL 阳性患者的 3 年 OS 显著低于阴性组（40.0% 对 85.7%；P=0.042）。3 年 DFS 也呈相同趋势，但差异无统计学意义（40.0% 对 75.0%；P=0.233）。
结论
CAR-T 治疗后序贯异基因 HSCT 可作为儿童 Ph 阳性 B-ALL 的安全有效治疗。
展开英文摘要原文
BACKGROUND
Philadelphia chromosome (Ph)-positive B-cell acute lymphoblastic leukemia (ALL) has a high complete remission (CR) rate, but relapse and prolonged measurable residual disease remain serious problems. We sought to describe the CR rate measurable residual disease negative rate and address the results and safety of pediatric patients who underwent after receiving chimeric antigen receptor (CAR) specific for CD19 (CAR-19) followed by hematopoietic stem cell transplantation (HSCT) for the treatment of Ph-positive ALL.
METHODS
A descriptive study was conducted at Peking University People's Hospital from September 2013 to January 2021. 13 patients with relapsed/refractory Ph-positive B-ALL who received CAR-T therapy followed by allo-HSCT were included. We concentrated on the overall patient survival and CR rate.
RESULTS
The median time between CAR-T therapy and allo-HSCT was 58 days. Among all the patients, the CR rate was 100%, the flow cytometry negativity rate was 84.62%, and the BCR-ABL negativity rate was 53.85% at 1 month after CAR-T infusion. All the patients achieved a major molecular response in 6 months after HSCT. After a median follow-up of 45 months, the 3-year OS rate was 66.7%, and the 3-year DFS rate was 61.5%. The 3-year OS rate of patients with BCR-ABL-positive pre-HSCT was significantly lower than that in the BCR-ABL-negative group (40.0% vs. 85.7%, P =0.042). Also, the same trend was observed for the 3-year DFS rate but did not differ significantly (40.0% vs. 75.0%, P =0.233).
CONCLUSIONS
CAR-T therapy followed by allo-HSCT can be a safe and effective treatment for Ph-positive B-ALL pediatric patients.
论文信息
- 作者
- Li Y、Hu GH、Xu LP、Zhang XH、Liu KY、Suo P、Wang Y、Cheng YF
- 第一作者单位
- Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, Chinese Academic of Medical Sciences, Beijing, China.China
- 文献类型
- 非美国政府资助研究
- 期刊
- Journal of pediatric hematology/oncology2024 Jul 1
- 原文标识
- PubMed 38652054 · DOI 10.1097/MPH.0000000000002861