Journal article

Venetoclax in combination with cytarabine with or without idarubicin in children with relapsed or refractory acute myeloid leukaemia: a phase 1, dose-escalation study.

Seth E Karol, Thomas B Alexander, Amit Budhraja, Stanley B Pounds, Kristin Canavera, Lei Wang, Joshua Wolf, Jeffery M Klco, Paul E Mead, Soumyasri Das Gupta, Su Y Kim, Ahmed Hamed Salem, Tammy Palenski, Norman J Lacayo, Ching-Hon Pui, Joseph T Opferman, Jeffrey E Rubnitz

The Lancet Oncology | Published : 2020


BACKGROUND: Outcomes for children with relapsed or refractory acute myeloid leukaemia remain poor. The BCL-2 inhibitor, venetoclax, has shown promising activity in combination with hypomethylating agents and low-dose cytarabine in older adults for whom chemotherapy is not suitable with newly diagnosed acute myeloid leukaemia. We aimed to determine the safety and explore the activity of venetoclax in combination with standard and high-dose chemotherapy in paediatric patients with relapsed or refractory acute myeloid leukaemia. METHODS: We did a phase 1, dose-escalation study at three research hospitals in the USA. Eligible patients were aged 2-22 years with relapsed or refractory acute myeloi..

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