In May 2022, the FDA approved Vidaza (azacitidine for injection; from Celgene) for the treatment of young patients with newly diagnosed juvenile myelomonocytic leukemia, a condition that can develop into myelodysplastic syndromes. Vidaza was previously approved for the treatment of patients with myelodysplastic syndromes.
This approval was based on results of a clinical trial that evaluated Vidaza in 18 pediatric patients with juvenile myelomonocytic leukemia. All the patients received Vidaza on days 1 to 7 of a 28-day cycle, for a minimum of 3 cycles and a maximum of 6 cycles. Patients were included in the study if they did not have disease progression or were not ready for a transplant between cycles 4 and 6.
Of the 18 patients in the study, half (9) of the patients had clinical responses to treatment with Vidaza. Of these 9 patients, 3 patients had a complete response (no sign of cancer) to therapy and 6 patients had a partial response. The average time to response was 1.2 months.
The majority (94%) of the patients received a transplant after treatment with Vidaza, and the average time to the transplant was 4.6 months.
The most common side effects reported with Vidaza in pediatric patients with juvenile myelomonocytic leukemia were fever, rash, upper respiratory tract infection, and anemia.
