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复发/难治性皮质(CD1a⁺)T 细胞急性淋巴细胞白血病儿童与成人患者的结局:SEHOP 与 PETHEMA 协作组的西班牙经验

英文原题:Outcomes of pediatric and adult patients with relapsed/refractory cortical (CD1a+) T-cell acute lymphoblastic leukemia. The Spanish experience from SEHOP and PETHEMA groups.

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Outcomes of pediatric and adult patients with relapsed/refractory cortical (CD1a+) T-cell acute lymphoblastic leukemia. The Spanish experience from SEHOP and PETHEMA groups.

PubMed 2026/03/24(内容时间) Ann Hematol Q3 · IF 2.3(JCR 2025)

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中文摘要

复发/难治性T细胞急性淋巴细胞白血病(R/R T-ALL)患者预后极差。CAR-T 细胞疗法正在这些患者中进行探索,并取得了令人鼓舞的结果。

具体而言,CD1a导向的CAR-T 细胞正在临床试验中用于治疗皮质T-ALL(CD1a+),但在该疗法之前,尚无关于该亚组患者结局的数据。这项回顾性观察性研究旨在描述R/R CD1a+ T-ALL患者的特征和结局。

我们纳入了2006年3月至2022年5月期间在西班牙25个中心诊断为R/R CD1a+ T-ALL的儿童和成人患者。研究的主要结局为总生存期(OS)。共纳入43例R/R CD1a+ T-ALL患者,其中28例成人和15例儿童。纳入时中位(范围)年龄为24岁(5-57),82.5%为男性。在中位(范围)随访7.0年(5.1-13.6)后,6例(14.0%)患者存活并处于完全缓解(CR),37例(86.0%)已死亡。5年OS为16%(95% CI;7-29%)。骨髓复发、首次CR与复发之间的间隔< 12个月,以及挽救治疗期间未接受异基因造血干细胞移植与更差的OS相关。

我们的结果证实,R/R CD1a+ T-ALL患者预后极差,凸显了这些患者对新治疗选择的需求。

展开英文摘要原文

Patients with relapsed/refractory T-cell acute lymphoblastic leukemia (R/R T-ALL) have very poor prognosis. CAR T-cell therapy is being explored in these patients with encouraging results. Specifically, CD1a-directed CAR T-cells are being explored in clinical trials to treat cortical T-ALL (CD1a+), but there are no data on outcome in this subgroup of patients before this therapy. This retrospective, observational study aimed to describe the characteristics and outcomes of patients with R/R CD1a + T-ALL.

We included pediatric and adult patients diagnosed with R/R CD1a + T-ALL in 25 sites of Spain between March 2006 and May 2022. The primary outcome of the study was overall survival (OS). Forty-three patients, 28 adults and 15 children, with R/R CD1a + T-ALL were included. Median (range) age at inclusion was 24 years (5 57), and 82. 5% were male.

After a median (range) follow-up of 7. 0 years (5. 1 13. 6), 6 (14. 0%) patients were alive and in complete remission (CR) and 37 (86. 0%) had died. Five-year OS was 16% (95% CI; 7 29%). Bone marrow relapse, an interval < 12 months between first CR and relapse, and lack of allogeneic hematopoietic stem cell transplantation during salvage treatment were associated with worse OS.

Our results confirm that patients with R/R CD1a + T-ALL have a very poor prognosis, highlighting the need for new treatment alternatives in these patients.

论文信息

作者
Rivera-Pérez C、Morgades M、Alonso-Saladrigues A、Montesinos P、Murciano T、Gil C、Adán R、Esteve J
第一作者单位
Stem Cell Transplant Department, Pediatric Cancer Center Barcelona (PCCB) - Hospital Sant Joan de D&#xe9;u de Barcelona, Barcelona, Spain.Spain
通讯作者单位
Leukemia and Lymphoma Department, Pediatric Cancer Center Barcelona (PCCB) - Hospital Sant Joan de D&#xe9;u de Barcelona, Barcelona, Spain. susana.rives@sjd.es.Spain
文献类型
多中心研究 · 观察性研究
期刊
Annals of hematology2026 Mar 24
原文标识
PubMed 41874722 · DOI 10.1007/s00277-026-06956-8