Azacitidine treatment for myeloid leukemia associated with Down syndrome: A nationwide retrospective study in Japan.
Shota KatoKentaro NakashimaGenki YamatoShoji SaitoYuichi TaneyamaNobuyuki YamamotoTakako MiyamuraKeisuke KatoYuya SatoAi YamadaTakahiro KamiyaTakuro NishikawaSuguru UemuraMasanori YoshidaHiroshi MirotakiKiminori TeruiTakashi TagaDaisuke HasegawaPublished in: Pediatric blood & cancer (2024)
Hypomethylating agent treatment for myeloid leukemia associated with Down syndrome (ML-DS) has been scarcely reported. Herein, we collected information on azacitidine treatment for ML-DS in Japan. Forty-eight cycles of azacitidine treatment were performed for 12 patients, including 11 relapsed or refractory (R/R) patients. In 40 cycles, azacitidine was used as monotherapy. No azacitidine-related death was observed. One cycle concurrently administered with methotrexate-based intrathecal therapy was discontinued due to toxicities. Only 4 of the 19 cycles given in non-remission achieved complete or partial remission. In conclusion, although most toxicities were acceptable, azacitidine monotherapy might be insufficient for R/R ML-DS cases.
Keyphrases
- acute myeloid leukemia
- end stage renal disease
- combination therapy
- ejection fraction
- chronic kidney disease
- newly diagnosed
- healthcare
- low dose
- clinical trial
- high dose
- cross sectional
- social media
- open label
- diffuse large b cell lymphoma
- ulcerative colitis
- disease activity
- patient reported
- replacement therapy
- drug induced
- chemotherapy induced