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

Franco Locatelli, Karin Belander Strålin, Irene Schmid, Julián Sevilla, Owen P Smith, Marry M van den Heuvel-Eibrink, Marco Zecca, Christian M Zwaan, Allison Gaudy, Meera Patturajan, Jennifer Poon, Mathew Simcock, Charlotte M Niemeyer

Onderzoeksoutput: Bijdrage aan tijdschriftArtikelpeer review

Samenvatting

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.

Originele taal-2Engels
Pagina's (van-tot)e30931
TijdschriftPediatric blood & cancer
Volume71
Nummer van het tijdschrift5
Vroegere onlinedatum3 mrt. 2024
DOI's
StatusGepubliceerd - mei 2024

Trefwoorden

  • MDS
  • azacitidine
  • hypomethylating agents
  • myelodysplastic syndromes
  • myeloid neoplasia
  • pediatrics

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