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

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 F...

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