title: Low dose decitabine in very high risk relapsed or refractory acute myeloid leukaemia in children and young adults.
abstract: 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 4 years) 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 2.5 cycles (range 1-4 cycles). Four patients received subsequent allogeneic stem cell transplant, and two remain in long-term CR.
authors: Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 452229, USA. christine.phillips@cchmc.org
authors: Christine L
authors: CL
authors: Phillips
authors: Stella M
authors: SM
authors: Davies
authors: Richard
authors: R
authors: McMasters
authors: Michael
authors: M
authors: Absalon
authors: Maureen
authors: M
authors: O'Brien
authors: Jun
authors: J
authors: Mo
authors: Randall
authors: R
authors: Broun
authors: Jeffrey A
authors: JA
authors: Moscow
authors: Teresa
authors: T
authors: Smolarek
authors: Ramiro
authors: R
authors: Garzon
authors: William
authors: W
authors: Blum
authors: Sebastian
authors: S
authors: Schwind
authors: Guido
authors: G
authors: Marcucci
authors: John P
authors: JP
authors: Perentesis
mesh_terms: D000293:Adolescent; D000328:Adult; D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001374:Azacitidine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D001853:Bone Marrow / Q000737:chemistry; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D000076123:DNA (Cytosine-5-)-Methyltransferase 1; D004248:DNA (Cytosine-5-)-Methyltransferases / Q000032:analysis; D000077209:Decitabine; D004341:Drug Evaluation; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000235:genetics / Q000601:surgery; D008297:Male; D035683:MicroRNAs / Q000032:analysis; D009363:Neoplasm Proteins / Q000032:analysis; D009503:Neutropenia / Q000139:chemically induced; D012334:RNA, Neoplasm / Q000032:analysis; D012074:Remission Induction; D016879:Salvage Therapy; D033581:Stem Cell Transplantation; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; C532045:MIRN29a microRNA, human; D035683:MicroRNAs; D009363:Neoplasm Proteins; D012334:RNA, Neoplasm; D000077209:Decitabine; D000076123:DNA (Cytosine-5-)-Methyltransferase 1; D004248:DNA (Cytosine-5-)-Methyltransferases; D001374:Azacitidine