阿扎胞苷成功维持了一名KMT2A重排急性淋巴细胞白血病婴儿的第二次缓解，该婴儿在非亲缘脐血移植后复发。
Azacitidine successfully maintained the second remission in an infant with KMT2A-rearranged acute lymphoblastic leukemia who relapsed after unrelated cord blood transplantation.
作者信息
Chijimatsu Ikue, Imanaka Yusuke, Tomizawa Daisuke, Eguchi Mariko, Nishimura Shiho, Karakawa Shuhei, Miki Mizuka, Hamamoto Kazuko, Fujita Naoto
机构信息
Department of Pediatrics, Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital, Hiroshima, Japan.
Department of Pediatrics, Hiroshima University Graduate School of Biomedical and Health Sciences, Hiroshima, Japan.
出版信息
Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26697. Epub 2017 Jul 4.
The outcome for infants with KMT2A (MLL)-rearranged acute lymphoblastic leukemia (MLL-r ALL) is dismal despite intensive therapy, including hematopoietic stem cell transplantation (HSCT). Epigenetic dysregulation is considered a key driver of MLL-r leukemogenesis, which theoretically supports the use of epigenetic modifiers as a treatment option. We report an infant MLL-r ALL case with post-HSCT relapse. After achieving a second remission, which was maintained for 10 months using only the DNA methyltransferase inhibitor, azacitidine, the patient successfully received the second HSCT. This report describes the clinical effectiveness of azacitidine for the treatment of infant MLL-r ALL.
尽管接受了包括造血干细胞移植（HSCT）在内的强化治疗，但患有KMT2A（MLL）重排急性淋巴细胞白血病（MLL-r ALL）的婴儿预后仍然很差。表观遗传失调被认为是MLL-r白血病发生的关键驱动因素，这在理论上支持使用表观遗传修饰剂作为一种治疗选择。我们报告了1例HSCT后复发的婴儿MLL-r ALL病例。在仅使用DNA甲基转移酶抑制剂阿扎胞苷维持第二次缓解10个月后，该患者成功接受了第二次HSCT。本报告描述了阿扎胞苷治疗婴儿MLL-r ALL的临床疗效。
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