1. Azacitidine treatment for myeloid leukemia associated with Down syndrome: A nationwide retrospective study in Japan.
- Author
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Kato S, Nakashima K, Yamato G, Saito S, Taneyama Y, Yamamoto N, Miyamura T, Kato K, Sato Y, Yamada A, Kamiya T, Nishikawa T, Uemura S, Tomizawa D, Moritake H, Terui K, Taga T, and Hasegawa D
- Subjects
- Humans, Male, Female, Retrospective Studies, Japan epidemiology, Child, Preschool, Child, Adolescent, Infant, Adult, Down Syndrome complications, Down Syndrome drug therapy, Azacitidine therapeutic use, Azacitidine adverse effects, Antimetabolites, Antineoplastic therapeutic use, Antimetabolites, Antineoplastic adverse effects, Leukemia, Myeloid drug therapy, Leukemia, Myeloid complications
- Abstract
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., (© 2024 Wiley Periodicals LLC.)
- Published
- 2024
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