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异基因造血干细胞移植治疗伴 E2A::HLF 融合基因儿童急性淋巴细胞白血病的疗效分析:病例系列

英文原题:Analysis of the therapeutic effect of allogeneic hematopoietic stem cell transplantation in the treatment of pediatric acute lymphoblastic leukemia with E2A::HLF fusion gene: a case series.

查看英文原题

Analysis of the therapeutic effect of allogeneic hematopoietic stem cell transplantation in the treatment of pediatric acute lymphoblastic leukemia with E2A::HLF fusion gene: a case series.

PubMed 2025/12/15(内容时间) Transl Pediatr Q2 · IF 2(JCR 2025)

分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。

研究概要

HSCT 前的 CAR-T 细胞治疗联合 HSCT 后的 pro-DLI 治疗,可能是改善这些儿科患者结局的有效策略。

中文摘要

E2A::HLF(TCF3::HLF)融合基因见于不足1%的B细胞急性淋巴细胞白血病(ALL)患者,主要发生于较大儿童和青少年。即使接受造血干细胞移植(HSCT),E2A::HLF融合型B-ALL患者结局通常仍很差。此类疾病中应用CAR-T 细胞和供者淋巴细胞输注(DLI)的报道很少。本病例系列重点评估CAR-T 和DLI治疗的安全性与疗效,以优化高危亚组治疗策略并改善生存。病例描述:回顾4例E2A::HLF融合型儿童B-ALL患者的临床病程。4例均按中国儿童白血病协作组(CCLG)2018-ALL高危分层方案治疗。病例1和病例4经强化治疗后仍未缓解,随后接受CD19-CAR-T 治疗。4例患者均在HSCT前达到完全缓解,骨髓微小残留病和E2A::HLF融合检测均阴性。病例3死于HSCT并发症。病例1和病例4在HSCT后接受DLI,目前仍存活。病例2在HSCT后复发;尽管后续接受CAR-T 和DLI治疗,仍因原发病再次复发而死亡。

HSCT前给予CAR-T 治疗并在HSCT后进行预防性DLI,可能是改善这类儿童患者结局的有效策略。

展开英文摘要原文

The fusion gene E2A::HLF ( TCF3::HLF ) is present in <1% of B-cell acute lymphoblastic leukemia (ALL) patients, mainly in older children and adolescents. Patients with E2A::HLF fusion B-ALL consistently have dismal outcomes, even after hematopoietic stem cell transplantation (HSCT). Chimeric antigen receptor T-cell (CAR-T) therapy and donor lymphocyte infusion (DLI) applied in this kind of disease have rarely been reported. In this case series, we will focus on the safety and effectiveness of CAR-T and DLI treatments. This may refine therapeutic strategies and improve survival for this high-risk subgroup. CASE DESCRIPTION: In this case series, we review the clinical course of 4 pediatric patients with E2A::HLF fusion B-ALL. All four patients treated with Chinese Children Leukemia Group (CCLG)-2018-ALL protocol based on high-risk stratification. Two patients (case 1 and case 4) maintained non-remission after intensive therapy and then accepted CD19-CAR-T therapy. All four patients achieved complete remission before HSCT and were negative for minimal residual disease and E2A::HLF fusion in the bone marrow. One patient (case 3) succumbed to complication of HSCT. Two patients (case 1 and case 4) who accepted DLI treatment after HSCT were still alive. One patient (case 2) relapsed after HSCT. Although accepting CAR-T and DLI treatments after HSCT, she died due to primary disease recurrent relapse.

The combination of CAR-T-cell therapy before HSCT and pro-DLI treatment after HSCT may represent an effective strategy to improve outcomes in these pediatric patients.

论文信息

作者
Jing Y、Wei A、Jia C、Yang J、Zhu G、Luo Y、Zhang K、Yang W
单位
Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Department of Stem cell Transplantation, Beijing Children's Hospital, Capital Medical University; National Center for Children's Health, Beijing, China.China
文献类型
病例报告
期刊
Translational pediatrics2025 Dec 31
原文标识
PubMed 41502902 · DOI 10.21037/tp-2025-631