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
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.
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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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。
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.
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