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-2 | Engels |
---|---|
Pagina's (van-tot) | e30931 |
Tijdschrift | Pediatric blood & cancer |
Volume | 71 |
Nummer van het tijdschrift | 5 |
Vroegere onlinedatum | 3 mrt. 2024 |
DOI's | |
Status | Gepubliceerd - mei 2024 |
Trefwoorden
- MDS
- azacitidine
- hypomethylating agents
- myelodysplastic syndromes
- myeloid neoplasia
- pediatrics