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CHRONIC MYELOGENOUS LEUKEMIA

Management of children and adolescents with chronic myeloid leukemia in chronic phase: International pediatric chronic myeloid leukemia expert panel recommendations

Leukemia volume 39, pages 779–791 (2025)Cite this article

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Abstract

The treatment strategy for children and adolescents with chronic myeloid leukemia in the chronic phase (CML-CP) has evolved from allogeneic hematopoietic stem cell transplantation (HSCT) to tyrosine kinase inhibitors (TKIs). With the advent of next-generation TKIs and new targeted therapies in the CML field, an international pediatric CML expert panel provides recommendations based on the medical literature (including previous pediatric guidelines), national standards, and treatment principles used in adults with CML-CP. Recommendations include diagnosis of the disease and details on managing the initial steps of care of children and adolescents with newly diagnosed CML-CP, including complications such as leukostasis. The treatment recommendations are based on the initiation of therapy with a first- or second-generation TKI according to the allocated European Treatment and Outcome Study (EUTOS) long-term survival score risk group of the patient. The subsequent steps are based on the results of recommended monitoring which can justify a switch to another TKI or a drug in development if there is resistance or toxicity. The panel also provides recommendations regarding the discontinuation criteria for TKIs in children and adolescents in sustained deep molecular response. Allogeneic HSCT is not recommended as the first-line of treatment for children with CML-CP but is to be considered in case of progression to the advanced phase or failure of several lines of treatment. The present treatment and management recommendations are intended to provide advice to clinicians in view of optimizing the care and the outcome of children and adolescents with CML-CP.

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Fig. 1: Algorithm of treatment recommendations for children and adolescents with chronic myeloid leukemia in the chronic phase.

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Acknowledgements

The authors gratefully acknowledge Professor Irene Roberts (emeritus professor of pediatric hematology, MRC Weatherall Institute of Molecular Medicine, Oxford University, United Kingdom) and Professor François Guilhot (emeritus professor of hematology, University of Poitiers, France) for helpful comments on the manuscript. The manuscript was reread and reviewed by Jeffrey Arsham, the American medical proofreader at the University Hospital of Poitiers, France.

Author information

Authors and Affiliations

  1. Inserm CIC 1402, University Hospital of Poitiers, Poitiers, France

    Frédéric Millot

  2. Department of Pediatric Hematology Oncology (T.A.O.), Aghia Sophia Children’s Hospital, Athens, Greece

    Mirella Ampatzidou

  3. Department of Pediatric Oncology, Tata Memorial Hospital, Homi Bhabha National Institute, Mumbai, India

    Nirmalya Roy Moulik

  4. Department of Paediatric Oncology/Haematology, Royal Marsden NHS Foundation Trust Sutton, Sutton, UK

    Sanjay Tewari

  5. Department of Pediatric Oncology, Children’s Cancer Hospital, National Cancer Institute, Cairo University, Cairo, Egypt

    Alaa Elhaddad & Mahmoud Hammad

  6. Department of Pediatrics and Adolescent Medicine, St. Anna Children’s Hospital, Medical University of Vienna, Vienna, Austria

    Herbert Pichler & Michael Dworzak

  7. St. Anna Children’s Cancer Research Institute (CCRI), Vienna, Austria

    Thomas Lion & Michael Dworzak

  8. Labdia Labordiagnostik, Vienna, Austria

    Thomas Lion

  9. 2nd Department of Pediatrics, School of Medicine, Faculty of Health Sciences, Aristotle University of Thessaloniki, AHEPA University Hospital, Thessaloniki, Greece

    Athanasios Tragiannidis

  10. Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan

    Haruko Shima

  11. State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin, China

    Wenbin An & Wenyu Yang

  12. Division of Pediatric Oncology and Hematology, Department of Pediatrics and Adolescent Medicine, University Hospital, Erlangen, Germany

    Axel Karow, Stephanie Sembill & Markus Metzler

  13. Department of Pediatrics, Lebanese American University Medical Center-Rizk Hospital, Beirut, Lebanon

    Roula Farah

  14. Department of Pediatric Hemato-Oncology, Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands

    Maaike Luesink

  15. Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium

    Barbara De Moerloose

  16. Department of Pediatric Hematology-Oncology, University Hospital Motol, Charles University, Prague, Czech Republic

    Petr Sedlacek

  17. Division of Hematology/Oncology/BMT, British Columbia Children’s Hospital Research Institute, Vancouver, BC, Canada

    Kirk R. Schultz

  18. Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University, Wroclaw, Poland

    Krzysztof Kalwak

  19. Department of Pediatric Blood and Marrow Transplantation, Princess Máxima Center, Utrecht, The Netherlands

    Birgitta Versluys

  20. Division of Haematology-Oncology, Department of Pediatrics, McMaster University, Hamilton, ON, Canada

    Uma Athale

  21. Division of Pediatric Hematology/Oncology/Stem Cell Transplant, Department of Pediatrics, Columbia University Irving Medical Center, New York, NY, USA

    Nobuko Hijiya

  22. Department of Pediatric Hematology and Oncology, Medical Faculty, Technical University, Dresden, Germany

    Meinolf Suttorp

Authors
  1. Frédéric Millot
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  2. Mirella Ampatzidou
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Contributions

FM, MS, and MM designed the project and defined items for the panel discussion. FM, MA, NRM, ST, AE, MH, HP, TL, AT, HS, WA, WY, AK, RF, ML, MD, SS, BDM, PS, KRS, KK, BV, UA, NH, MM, and MS contributed to the literature review, to the discussion and the detailed recommendations. FM drafted the manuscript. All authors revised and approved the final version of the manuscript.

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Correspondence to Frédéric Millot.

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Millot, F., Ampatzidou, M., Moulik, N.R. et al. Management of children and adolescents with chronic myeloid leukemia in chronic phase: International pediatric chronic myeloid leukemia expert panel recommendations. Leukemia 39, 779–791 (2025). https://doi.org/10.1038/s41375-025-02543-4

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