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. 2024 Oct;71(10):e31244.
doi: 10.1002/pbc.31244. Epub 2024 Aug 4.

Azacitidine treatment for myeloid leukemia associated with Down syndrome: A nationwide retrospective study in Japan

Shota Kato  1 , Kentaro Nakashima  2 , Genki Yamato  3 , Shoji Saito  4 , Yuichi Taneyama  5 , Nobuyuki Yamamoto  6 , Takako Miyamura  7 , Keisuke Kato  8 , Yuya Sato  9 , Ai Yamada  10 , Takahiro Kamiya  11 , Takuro Nishikawa  12 , Suguru Uemura  13 , Daisuke Tomizawa  14 , Hiroshi Moritake  10 , Kiminori Terui  15 , Takashi Taga  16 , Daisuke Hasegawa  17
Affiliations

Azacitidine treatment for myeloid leukemia associated with Down syndrome: A nationwide retrospective study in Japan

Shota Kato et al. Pediatr Blood Cancer. 2024 Oct.

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.

Keywords: Azacitidine; hypomethylating agents; myeloid leukemia associated with Down syndrome; novel treatment.

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References

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