Article
Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial.
Blood advances - 27 Jul 2021
Niemeyer Charlotte M, Flotho Christian, Lipka Daniel B, Starý Jan, Rössig Claudia, Baruchel André, Klingebiel Thomas, Micalizzi Concetta, Michel Gérard, Nysom Karsten, Rives Susana, Schmugge Liner Markus, Zecca Marco, Schönung Maximilian, Baumann Irith, Nöllke Peter, Benettaib Bouchra, Biserna Noha, Poon Jennifer, Simcock Mathew, Patturajan Meera, Menezes Daniel, Gaudy Allison, van den Heuvel-Eibrink Marry M, Locatelli Franco
Abstract excerpt
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy for most children with juvenile myelomonocytic leukemia (JMML). Novel therapies controlling the disorder prior to HSCT are needed. We conducted a phase 2, multicenter, open-label study to evaluate the safety and antileukemic activity of azacitidine monotherapy prior to HSCT in newly diagnosed JMML patients. Eighteen patients...
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