Efficacy and safety of azacitidine in pediatric patients with newly diagnosed advanced myelodysplastic syndromes before hematopoietic stem cell transplantation in the AZA‐JMML‐001 trial

Abstract: Here we report efficacy, pharmacokinetics, and safety data obtained in treatment-naive, pediatric patients with newly diagnosed advanced MDS receiving azacitidine in the AZA-JMML-001 study. The primary endpoint was response rate (proportion of patients with complete response [CR], partial response [PR], or marrow CR, sustained for ≥4 weeks). Of the 10 patients enrolled, one had an unconfirmed marrow CR and none had confirmed responses after three cycles; the study was therefore closed after stage 1. Azacitidine was well tolerated. The lack of efficacy of azacitidine in pediatric patients with newly diagnosed advanced MDS highlights the need for effective new treatments in these patients

Object Details

• Location: Deutsche Nationalbibliothek Frankfurt am Main
• Extent: Online-Ressource
• Language: Englisch
• Notes: Pediatric blood & cancer. - 71, 5 (2024) , e30931, ISSN: 1545-5017

Contributors, Places and Time

• Event: Veröffentlichung
• (where): Freiburg
• (who): Universität
• (when): 2024
• Creator: Locatelli, Franco ; Strålin, Karin Belander ; Schmid, Irene ; Sevilla, Julian ; Smith, Owen P. ; Heuvel-Eibrink, Marry M. van den ; Zecca, Marco ; Zwaan, Christian Michel ; Gaudy, Allison ; Patturajan, Meera ; Poon, Jennifer ; Simcock, Mathew ; Niemeyer, Charlotte

Further information

• DOI: 10.1002/pbc.30931
• URN: urn:nbn:de:bsz:25-freidok-2473015
• Rights: Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
• Last update: 14.08.2025, 10:59 AM CEST