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

• Published in: Pediatric blood & cancer Vol. 71; no. 5; pp. e30931 - n/a
• Main Authors: Locatelli, Franco, Strålin, Karin Belander, Schmid, Irene, Sevilla, Julián, Smith, Owen P., den Heuvel‐Eibrink, Marry M., Zecca, Marco, Zwaan, Christian M., Gaudy, Allison, Patturajan, Meera, Poon, Jennifer, Simcock, Mathew, Niemeyer, Charlotte M.
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.05.2024
• Subjects: azacitidine; Hematopoietic stem cells; hypomethylating agents; MDS; Myelodysplastic syndrome; myelodysplastic syndromes; myeloid neoplasia; Myelomonocytic leukemia; Patients; Pediatrics; Pharmacokinetics; Stem cell transplantation; hypomethylating agents; MDS; myeloid neoplasia; azacitidine; myelodysplastic syndromes; pediatrics
• ISSN: 1545-5009; 1545-5017; 1545-5017
• DOI: 10.1002/pbc.30931

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.