Azacitidine Approved for Treatment of Pediatric Patients with Newly Diagnosed Myelomonocytic Leukemia

Myelomonocytic leukemia cells.

The FDA has approved azacitidine (Vidaza®, Celgene) for patients with newly diagnosed juvenile myelomonocytic leukemia (JMML), prior to allogeneic hematopoietic stem cell transplantation (HSCT).

“JMML is a rare, unique myeloproliferative/myelodysplastic neoplasia of early childhood driven by canonical Ras-pathway mutations in PTPN11, NRAS, KRAS, NF1, or CBL,” wrote Charlotte M. Niemeyer, MD, Professor and Medical Director in the Department of Pediatric Hematology and Oncology at Medical Center Freiburg of the University of Freiburg in Germany, and colleagues, in their published results of the AZA-JMML-001 trial (NCT02447666), on which the approval was based. “Novel therapies controlling the disorder prior to HSCT are needed.”

The phase 2, open-label, international, multicenter trial enrolled 18 pediatric patients, between the ages of one month and <18 years, with newly diagnosed JMML who had not undergone prior HSCT. Upon enrollment, each patient was given 75 mg/m2 of azacitidine via IV for 10 to 40 minutes on Days 1 through 7 during a 28-day cycle, for a minimum of 3 and maximum of 6 cycles. Patients who were <10 kg body weight or <1 year old were treated with 2.5 mg/kg IV of azacitidine at the same rate and cycle parameters. If a patient had disease progression or was ready for HSCT between Cycles 4 and 6, they would be discontinued from treatment with azacitidine. The primary end points were clinical complete remission or clinical partial remission at Cycle 3 Day 28, with secondary end points of leukemia-free survival and overall survival.

At the succession of the third cycle, a total of nine patients (50%) had a confirmed clinical response, with three of them having clinical complete remission and six having clinical partial remission after a median time to response of 1.2 months. At a median follow-up of 23.8 months, 17 patients (94%) survived and underwent HSCT, with 14 patients (82%) experiencing leukemia-free survival. At a median follow-up of 26.9 months, 16 patients (90%) were reported for overall survival.

The most common adverse reactions experienced by >30% pediatric patients with JMML receiving azacitidine were pyrexia, rash, upper respiratory tract infection, and anemia.

“Although the long-term advantage of azacitidine therapy remains to be fully assessed, this prospective clinical trial has shown that azacitidine therapy prior to HSCT is a safe and efficacious option for patients with JMML,” concluded Dr. Niemeyer and colleagues in their report.

The recommended dose for patients less than one year of age or those with less than 10 kg body weight is 2.5 mg/kg via IV. For those greater than one year old and above 10 kg body weight, the recommended dose is 75 mg/m2 via IV.

For More Information

Niemeyer CM, Flotho C, Lipka DB, et al (2021). Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial. Blood Adv, 5(14):2901-2908. DOI:10.1182/bloodadvances.2020004144 (2019). Study with azacitidine in pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (MDS) and juvenile myelomonocytic leukemia (JMML). NLM identifier: NCT02447666.

Vidaza® (azacitidine) prescribing information (2021). Available at:

US Food and Drug Administration (2022). FDA approves azacitidine for newly diagnosed juvenile myelomonocytic leukemia. Available at:

Image Credit: Dr Erhabor Osaro. Licensed under CC BY-SA 3.0

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