Azacitidine Approved for Pediatric Myelomonocytic Leukemia

The U.S. Food and Drug Administration (FDA) has approved azacitidine for pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).

The approval was based on data from the AZA-JMML trial, an international, multicenter, open-label study. The study included 18 patients with JMML treated with intravenous azacitidine daily on days 1-7 of a 28-day cycle for a minimum of three cycles and a maximum of six cycles, provided patients did not have disease progression or were ready for hematopoietic stem cell transplantation (HSCT) between cycles four and six.

The main efficacy outcome measures were clinical complete remission (cCR) or clinical partial remission (cPR) according to the International JMML response criteria at three months (cycle three, day 28). Responses must have been sustained for at least four weeks either in the four-week period preceding or succeeding cycle three, day 28.

Half of patients had confirmed clinical response. Of these, three had cCR and six had cPR. Median time to response was 1.2 months. The majority (94%) of patients underwent HSCT, and the median time to HSCT was 4.6 months.

Most common adverse reactions (>30%) occurring in pediatric patients with JMML were pyrexia, rash, upper respiratory tract infection, and anemia.

The recommended dose for patients aged 1 month to <1 year or weighing less than 10 kg is 2.5 mg/kg. The recommended dose for patients aged ≥1 and weighing ≥10 kg is 75 mg/m².

Source: FDA Press Release, May 2022