Azacitidine successfully maintained the second remission in an infant with KMT2A-rearranged acute lymphoblastic leukemia who relapsed after unrelated cord blood transplantation

作者：Chijimatsu Ikue^*; Imanaka Yusuke; Tomizawa Daisuke; Eguchi Mariko; Nishimura Shiho; Karakawa Shuhei; Miki Mizuka; Hamamoto Kazuko; Fujita Naoto

来源：Pediatric Blood and Cancer, 2017, 64(12): e26697.

DOI：10.1002/pbc.26697

• acute lymphoblastic leukemia
• azacitidine
• KMT2A
• MLL
• infant

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.

• 出版日期2017-12