FDA Grants Azacitidine Approval For Pediatric Patients With Myelomonocytic Leukemia


Azacitidine (Vidaza) is now FDA approved for pediatric patients with newly diagnosed myelomonocytic leukemia.

The FDA has approved azacitidine (Vidaza) to treat pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).1,2

The regulatory decision is supported by data from the international, multicenter, open-label AZA-JMML-001 (NCT02447666), in which investigators examined the safety and efficacy of azacitidine vs historical controls in pediatric patients with newly diagnosed myelodysplastic syndrome or JMML before hematopoietic stem cell transplantation (HSCT).

A total of 18 study participants were administered intravenous azacitidine at a daily dose of 75 mg/m2 on days 1 through 7 of a 28-day treatment cycle for a minimum of 3 cycles and a maximum of 6 cycles. The primary end point of the trial was response rate, which was comprised of complete remission (cCR) or clinical partial remission (cPR) per International JMML response criteria, at 3 months.

Results showed that 50% of patients (95% CI, 26%-74%) experienced confirmed clinical responses with the drug. Of these patients, 3 achieved a cCR and 6 experienced a cPR.

Moreover, the median time to response was 1.2 months (range, 0.95-1.87). Ninety-four percent of patients went on to receive HSCT, and the median time to transplant was 4.6 months (range, 2.8-19).

Prior data indicated that 38% of those who required platelet transfusion were free of transfusion following the third cycle of therapy. Of the 17 patients who went on to undergo HSCT, 82% were leukemia free at a median follow-up of 23.8 months (range, 7.0-39.3).

Regarding safety, the most common effects included pyrexia, rash, upper respiratory tract infection, and anemia.


  1. FDA approves azacitidine for newly diagnosed juvenile myelomonocytic leukemia. News release. FDA. May 20, 2022. Accessed May 23, 2022. https://bit.ly/3Lt1VrL
  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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