Azacitidine is approved by FDA for newly diagnosed juvenile myelomonocytic leukemia

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.

View full prescribing information for Vidaza.

Nishant Mittal (PhD)

+ postsBio ⮌

Dr. Nishant Mittal is a highly accomplished researcher with over 13 years of experience in the fields of cardiovascular biology and cancer research. His career is marked by significant contributions to stem cell biology, developmental biology, and innovative research techniques.

Research Highlights

Dr. Mittal's research has focused on several key areas:

1) Cardiovascular Development and Regeneration: He studied coronary vessel development and regeneration using zebrafish models1.

2) Cancer Biology: At Dartmouth College, he developed zebrafish models for studying tumor heterogeneity and clonal evolution in pancreatic cancer.
3) Developmental Biology: His doctoral work at Keio University involved identifying and characterizing medaka fish mutants with cardiovascular defects.

4) Stem Cell Research: He investigated the effects of folic acid on mouse embryonic stem cells and worked on cryopreservation techniques for hematopoietic stem cells.

Publications and Presentations

Dr. Mittal has authored several peer-reviewed publications in reputable journals such as Scientific Reports, Cardiovascular Research, and Disease Models & Mechanisms1. He has also presented his research at numerous international conferences, including the Stanford-Weill Cornell Cardiovascular Research Symposium and the Weinstein Cardiovascular Development Conference.

In summary, Dr. Nishant Mittal is a dedicated and accomplished researcher with a strong track record in cardiovascular and cancer biology, demonstrating expertise in various model systems and a commitment to advancing scientific knowledge through innovative research approaches.

• Nishant Mittal (PhD)

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  Brentuximab vedotin with lenalidomide and rituximab is approved by the USFDA for relapsed or refractory large B-cell lymphoma
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  Mirdametinib is approved by the USFDA for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection
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  Datopotamab deruxtecan-dlnk is approved by the USFDA for unresectable or metastatic, HR-positive, HER2-negative breast cancer
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  Remestemcel-L-rknd is approved by the USFDA for steroid-refractory acute graft versus host disease in pediatric patients