Juvenile myelomonocytic leukemia

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Section editor
David Noyd, MD, MPH
University of Washington
Seattle, WA, USA

1 regimens on this page
1 variants on this page

First-line therapy

Azacitidine monotherapy


Study Dates of enrollment Evidence
Niemeyer et al. 2021 (AZA-JMML-001) 2015-2017 Phase 2, fewer than 20 pts (RT)


  • Azacitidine (Vidaza) by the following age- and weight-based criteria:
    • Less than 10 kg or younger than 1 year old: 2.5 mg/kg IV over 10 to 40 minutes once per day on days 1 to 7
    • 10 kg or more and 1 year old or older: 75 mg/m2 IV once per day on days 1 to 7

28-day cycle for 3 to 6 cycles


  1. AZA-JMML-001: Niemeyer CM, Flotho C, Lipka DB, Starý J, Rössig C, Baruchel A, Klingebiel T, Micalizzi C, Michel G, Nysom K, Rives S, Schmugge Liner M, Zecca M, Schönung M, Baumann I, Nöllke P, Benettaib B, Biserna N, Poon J, Simcock M, Patturajan M, Menezes D, Gaudy A, van den Heuvel-Eibrink MM, Locatelli F. Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial. Blood Adv. 2021 Jul 27;5(14):2901-2908. link to original article link to PMC article contains dosing details in manuscript PubMed NCT02447666