Low dose decitabine in very high risk relapsed or refractory acute myeloid leukaemia in children and young adults

作者：Phillips Christine L^*; Davies Stella M; McMasters Richard; Absalon Michael; O'Brien Maureen; Mo Jun; Broun Randall; Moscow Jeffrey A; Smolarek Teresa; Garzon Ramiro; Blum William; Schwind Sebastian; Marcucci Guido; Perentesis John P

来源：British Journal of Haematology, 2013, 161(3): 406-410.

DOI：10.1111/bjh.12268

• paediatric
• relapsed acute myeloid leukaemia
• decitabine
• acute myeloid leukaemia
• epigenetic

Low-dose decitabine has encouraging activity and tolerability in adults with acute myeloid leukaemia (AML), but paediatric experience is lacking. We report our retrospective experience with decitabine in eight children and young adults (median age 4years) with refractory/relapsed AML, who had failed multiple regimens or were not candidates for standard retrieval regimens due to prior toxicities. Three of eight patients (38%) had complete response (CR; 1 each of CR, CR with incomplete platelet recovery and CR with incomplete count recovery). Best responses were observed after a median of 2 center dot 5 cycles (range 14 cycles). Four patients received subsequent allogeneic stem cell transplant, and two remain in long-term CR.

• 出版日期2013-5