Azacitidine treatment for myeloid leukemia associated with Down syndrome: A nationwide retrospective study in Japan-Reference-Cited by-同舟云学术

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

Published:2024-08-04 Issue:10 Volume:71 Page:
ISSN:1545-5009
Container-title:Pediatric Blood & Cancer
language:en
Short-container-title:Pediatric Blood & Cancer

Author:

Kato Shota¹^ORCID,Nakashima Kentaro²^ORCID,Yamato Genki³^ORCID,Saito Shoji⁴^ORCID,Taneyama Yuichi⁵,Yamamoto Nobuyuki⁶^ORCID,Miyamura Takako⁷^ORCID,Kato Keisuke⁸^ORCID,Sato Yuya⁹^ORCID,Yamada Ai¹⁰,Kamiya Takahiro¹¹,Nishikawa Takuro¹²^ORCID,Uemura Suguru¹³,Tomizawa Daisuke¹⁴^ORCID,Moritake Hiroshi¹⁰^ORCID,Terui Kiminori¹⁵^ORCID,Taga Takashi¹⁶^ORCID,Hasegawa Daisuke¹⁷^ORCID

Affiliation:

1. Department of Pediatrics, Graduate School of Medicine The University of Tokyo Tokyo Japan

2. Department of Pediatrics, Graduate School of Medical Sciences Kyushu University Fukuoka Japan

3. Department of Pediatrics Gunma University Graduate School of Medicine Maebashi Japan

4. Department of Pediatrics Shinshu University School of Medicine Matsumoto Japan

5. Department of Hematology/Oncology Chiba Children's Hospital Chiba Japan

6. Department of Pediatrics Kobe University Graduate School of Medicine Kobe Japan

7. Department of Pediatrics Osaka University Graduate School of Medicine Suita Japan

8. Division of Pediatric Hematology and Oncology Ibaraki Children's Hospital Mito Japan

9. Department of Pediatrics Dokkyo Medical University Tochigi Japan

10. Division of Pediatrics, Faculty of Medicine University of Miyazaki Miyazaki Japan

11. Department of Pediatrics and Developmental Biology, Graduate School of Medical and Dental Sciences Tokyo Medical and Dental University Tokyo Japan

12. Department of Pediatrics, Graduate School of Medical and Dental Sciences Kagoshima University Kagoshima Japan

13. Department of Hematology and Oncology Kobe Children's Hospital Kobe Japan

14. Division of Leukemia and Lymphoma, Children's Cancer Center National Center for Child Health and Development Tokyo Japan

15. Department of Pediatrics Hirosaki University Graduate School of Medicine Hirosaki Japan

16. Department of Pediatrics Shiga University of Medical Science Otsu Japan

17. Department of Pediatrics St. Luke's International Hospital Tokyo Japan

Abstract

AbstractHypomethylating 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.

Funder

Japan Agency for Medical Research and Development

Publisher

Wiley

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/pbc.31244

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