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
先天性 KMT2A-R B 淋巴细胞白血病长期生存者，伴持续性骨髓 MRD 阳性及多次 CNS 复发
英文原题：A long-term survivor of congenital KMT2A-R B-lymphoblastic leukemia with persistently positive bone marrow MRD and multiple CNS relapses.
查看英文原题
A long-term survivor of congenital KMT2A-R B-lymphoblastic leukemia with persistently positive bone marrow MRD and multiple CNS relapses.
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中文摘要
在此，我们描述一例在出生后第4天偶然诊断为先天性KMT2A重排（KMT2A-r）B细胞ALL的婴儿。他在出生后第5天接受了第一剂鞘内甲氨蝶呤，并在出生后第6天开始诱导全身治疗。他在诱导结束时达到形态学缓解，但骨髓呈阳性。微小残留病（MRD）为1.4%。在巩固和免疫治疗后，他出现了孤立性CNS疾病。在8个月大时，他接受了造血干细胞移植（HSCT）。在14个月大时，他出现了髓内和CNS复发，并在16个月大时接受了CD19 CAR-T 治疗。在6岁时，他仍处于缓解状态，伴有可耐受的发育迟缓以及良好的生活质量。
展开英文摘要原文
Here we describe the case of an infant incidentally diagnosed with congenital KMT2A-rearranged (KMT2A-r) B-cell ALL on Day of Life 4. He received the first dose of intrathecal methotrexate on DOL 5, and induction systemic therapy on DOL 6. He demonstrated morphologic remission at the end of induction but had positive bone marrow. Minimal residual disease (MRD) was 1.
4%. He experienced isolated CNS disease after consolidation and immunotherapy. At 8 months of age he underwent hematopoietic stem cell transplantation (HSCT). At 14 months of age he had medullary and CNS relapse, and at 16 months of age underwent CD19 CAR-T therapy. At 6 years of age he remains in remission with tolerable developmental delays and a good quality of life.
论文信息
- 作者
- Arabatzis TJ、Desai B、Baril S、Rasul S、Tekle-Yohannes G、Miller MA
- 第一作者单位
- Department of Internal Medicine-Pediatrics, Janet Weis Children's Hospital, Danville, PA, USA.United States
- 通讯作者单位
- Department of Pediatric Hematology and Oncology, Janet Weis Children's Hospital, 100 N Academy Ave, Danville, PA, 17822, USA. mamiller2@geisinger.edu.United States
- 文献类型
- 病例报告
- 期刊
- BMC pediatrics2025 Feb 28
- 原文标识
- PubMed 40016709 · DOI 10.1186/s12887-024-05093-3
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