Favorable outcomes of de novo advanced phases of pediatric chronic myeloid leukemia in the tyrosine kinase inhibitor era
Chronic myeloid leukemia (CML) is a rare disease during childhood, and accelerated phase (AP) and blast phase (BP) CML, also called advanced phases, are even rarer. We retrospectively collected and analyzed clinical data of children younger than 20 years with de novo advanced-phase CML between 1996 and 2017 in Japan. Median follow-up time was 8.9 years for AP-CML (n = 15) and 3.7 years for BP-CML (n = 32). The 5-year overall survival (OS) was 93.3% for AP-CML, and 100.0% for patients who received tyrosine kinase inhibitors (TKIs) in first-line therapy (n = 10). Four of the ten patients who received TKIs in first-line therapy remained in molecular remission without transplantation (median follow-up 5.5 years). The 5-year OS of patients with BP-CML was 79.0%, and most patients received chemotherapy before transplantation, with regimen selection based on blast immunophenotype. Furthermore, among patients who received transplantation after TKI therapy, the 5-year OS was 100.0% for AP and 84.8% for BP. In conclusion, our study confirmed excellent outcomes in children with de novo advanced-phase CML, especially in the TKI-era.
Authors
- Chikako Tono
- Toshihiko Imamura (ORCID: https://orcid.org/0000-0002-5727-4470)
- Dai Keino (ORCID: https://orcid.org/0000-0003-0312-1636)
- Masaki Ito (ORCID: https://orcid.org/0000-0002-8032-7198)
- Yukari Sakurai
- Keisuke Kato (ORCID: https://orcid.org/0000-0002-0135-5431)
- Hiroyuki Shimada (ORCID: https://orcid.org/0000-0002-5168-3222)
- Katsuyoshi Koh (ORCID: https://orcid.org/0000-0002-0476-4978)
- Hisashi Ishida (ORCID: https://orcid.org/0000-0002-3391-8403)
- Toshihide Yoshikawa (ORCID: https://orcid.org/0000-0003-3999-9379)
- Takuya Ichimura (ORCID: https://orcid.org/0000-0002-7477-8689)
- Yuko Osugi
- Haruko Shima (ORCID: https://orcid.org/0000-0003-3432-8832)
- Shosuke Sunami
- Akihiko Tanizawa (ORCID: https://orcid.org/0000-0002-0990-2508)
- Yuki Yuza (ORCID: https://orcid.org/0009-0003-6475-3394)
- Akihiro Watanabe
- Yuri Okimoto
- Kunihiro Shinoda (ORCID: https://orcid.org/0009-0009-8656-6673)
Institutions
- University of Fukui (JP)
- Hirosaki University (JP)
- Asahikawa Medical University (JP)
- Yamaguchi University (JP)
- Keio University (JP)
- Kyoto Prefectural University of Medicine (JP)
- Niigata Cancer Center Hospital (JP)
- Saitama Children's Medical Center (JP)
- Osaka City General Hospital (JP)
- Okayama University Hospital (JP)
- Gifu Municipal Hospital (JP)
- Kanagawa Children's Medical Center (JP)
- Chiba Hospital (JP)
- Japanese Red Cross Narita Hospital (JP)
- Tokyo Metropolitan Children's Medical Center (JP)
- Ibaraki Children's Hospital (JP)
- Soma Central Hospital (JP)
Publication Details
- Journal
- International Journal of Hematology
- Published
- 2025-03-15
- DOI
- https://doi.org/10.1007/s12185-025-03953-x
- Primary Topic
- Chronic Myeloid Leukemia Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Japan Agency for Medical Research and Development
- Ministry of Health, Labour and Welfare
- University of Fukui