Valeria Ceolin, Manuela Spadea, Vincenzo Apolito, Francesco Saglio, Franca Fagioli
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引用次数: 0
Abstract
The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last decades. However, at relapse, overall survival (OS) ∼40% to 50% and is even lower for patients with chemorefractory disease. Effective and less-toxic therapies are urgently needed for these children. In the last years, immune-directed therapies such as chimeric antigen receptor (CAR)-T cells were introduced, which showed outstanding clinical activity against B-cell malignancies. CART therapies are being developed for AML on the basis of the results obtained for other hematologic malignancies. The biggest challenge of CART therapy for AML is to identify a specific target antigen, since antigens expressed in AML cells are usually shared with healthy hematopoietic stem cells. An overview of prospects of CART in pediatric AML, focused on the common antigens targeted by CART in AML that have been tested or are currently under investigation, is provided in this manuscript.
过去几十年来，急性髓性白血病（AML）患儿的预后逐步改善。然而，复发时的总生存率（OS）仅为40%至50%，化疗难治性疾病患者的总生存率甚至更低。这些儿童迫切需要有效且毒性较低的疗法。近年来，嵌合抗原受体（CAR）-T 细胞等免疫导向疗法被引入临床，对 B 细胞恶性肿瘤显示出卓越的临床活性。在其他血液系统恶性肿瘤研究成果的基础上，目前正在开发针对急性髓细胞白血病的CART疗法。CART疗法治疗急性髓细胞性白血病的最大挑战是确定特定的靶抗原，因为急性髓细胞性白血病细胞表达的抗原通常与健康的造血干细胞共享。本手稿概述了CART在小儿急性髓细胞性白血病中的应用前景，重点介绍了CART在急性髓细胞性白血病中靶向的常见抗原，这些抗原已经过测试或正在研究中。
期刊介绍：
Journal of Pediatric Hematology/Oncology (JPHO) reports on major advances in the diagnosis and treatment of cancer and blood diseases in children. The journal publishes original research, commentaries, historical insights, and clinical and laboratory observations.
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