FDA Approved Azacitidine for Newly Diagnosed Juvenile Myelomonocytic Leukemia

Based on the results of the AZA-JMML-001 trial, the FDA has approved azacitidine for use in newly diagnosed juvenile myelomonocytic leukemia.

The FDA has approved azacitidine (Vidaza) for the treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).1

Results of the multicenter, open-label phase 2 AZA-JMML-001 trial (NCT02447666) which evaluated safety and efficacy of azacitidine against historical controls in pediatric patients with newly diagnosed advanced myelodysplastic syndrome or juvenile myelomonocytic leukemia before hematopoietic stem cell transplantation (HSCT) served as the basis for the approval.

In total, 18 patients were enrolled from September 2015 to November 2017 and were treated with once-daily intravenous azacitidine at 75 mg/m2 administered between 10 to 40 minutes on days 1 to 7 of a 28-day cycle for a minimum of 3 cycles and a maximum of 6. The primary end point was response rate, composed of clinical complete remission (cCR) or clinical partial remission (cPR) according to the International JMML response criteria, at 3 months.2

After 3 cycles, 50% (95% CI, 26%-74%) of patients were in cPR (n = 6) or cCR (n = 3). Median time to response was 1.2 months (range, 0.95-1.87). Most patients (94%) went on to receive HSCT, with a median time to transplant of 4.6 months (range, 2.8-19.0).

Previously reported data in 18 patients indicated that those who needed platelet transfusion (38%) were free of transfusion after the third cycle of therapy. Of the 17 patients who went on to receive HSCT, 14 (82%) were leukemia free at a median follow-up of 23.8 months (range, 7.0-39.3).

All patients experienced adverse events (AEs), with 10 patients (55.6%) experiencing toxicity related to treatment. Treatment-related AEs occurring in 2 (11%) patients each included anemia, thrombocytopenia, neutropenia, and constipation. One-third (33%) of patients had at least 1 grade 3 or 4 treatment-related AE. Only 1 event of treatment discontinuation was reported and was linked with a serious AE of abdominal pain that was not deemed to be treatment related.

The FDA-recommended dose for patients aged 1 month to less than 1 year or those weighing less than 10 kg is 2.5 mg/kg. Those who are over 1 year of age and who weigh at least 10 kg can be treated with the regimen outlined in the trial.


  1. FDA approves azacitidine for newly diagnosed juvenile myelomonocytic leukemia. FDA. May 20, 2022. Accessed May 20, 2022. https://bit.ly/3wEhxmI
  2. Niemeyer CM, Flotho C, Lipka DB, et al. Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial. Blood Adv. 2021;5(14):2901-2908. doi:10.1182/bloodadvances.2020004144
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