June 2022: The FDA has approved the drug azacitidine (Vidaza, Celgene Corp.) for children with newly diagnosed juvenile myelomonocytic leukaemia (JMML).
The pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to hematopoietic stem cell transplantation (HSCT) in 18 paediatric patients with JMML were evaluated in AZA-JMML-001 (NCT02447666), an international, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to HSCT in 18 paediatric patients with Patients were given intravenous azacitidine daily on Days 1-7 of a 28-day cycle for a minimum of 3 cycles and a maximum of 6 cycles if disease progression or readiness for HSCT did not occur between Cycles 4 and 6.
Clinical complete remission (cCR) or clinical partial remission (cPR) according to the International JMML response criteria at 3 months were the key efficacy outcome measures (Cycle 3, Day 28). Responses must have been sustained for at least four weeks in the four weeks preceding or following Cycle 3, Day 28. Clinical responses were confirmed in 9 individuals (50 percent, 95 percent CI: 26, 74). Three of the nine patients had cCR and six had cPR. The average response time was 1.2 months (range 0.95-1.87 months). The median duration to HSCT was 4.6 months, with 94 percent of patients undergoing the procedure (range 2.8-19 months).
Pyrexia, rash, upper respiratory tract infection, and anaemia were the most prevalent adverse events (>30%) in paediatric patients with JMML.
The suggested dose for patients aged one month to one year or weight less than ten kilogrammes is 2.5 mg/kg. 75 mg/m2 is the suggested amount for patients under the age of one and weighing less than ten kilogrammes.