Children with chronic-phase chronic myeloid leukemia: characteristics and outcomes from the International Registry of Childhood CML

Millot F, De Keizer J, Metzler M, Kalwak K, Günes AM, De Moerloose B, Luesink M, Honrubia AM, Sedlacek P, Yajima J, Ampatzidou M, Dworzak M, Lausen B, Borisevich M, Li CK, Kolenova A, Jakovljevic G, Farah R, Durocher L, Baruchel A, Suttorp M (2026)

Publication Type: Journal article

Publication year: 2026

Journal

Book Volume: 111

Pages Range: 1280-1288

Journal Issue: 4

DOI: 10.3324/haematol.2025.288283

Abstract

Five hundred and thirty-five patients (median age 12.2 years; range, 8 months to 18 years; N=303 males, N=232 females) diagnosed with chronic myeloid leukemia (CML) in chronic phase were registered in the International Registry of Childhood CML (clinicaltrials gov. Identifier: NCT01281735). Clinical signs of leukostasis or bleeding were observed in 16.5% and 20% of the patients, respectively. The spleen was palpable in 76% of patients with a median of 9 cm (range, 1-32) below the costal margin. The median leukocyte count was 222x109/L (interquartile range, 95-353). First-line therapy consisted of imatinib in 482 children. Among them, 40% remained on imatinib with a median follow-up 3.8 years (95% confidence interval [CI]: 3.2- 4.3 years); 3.1% died and 3.9% progressed to advanced-phase disease. The overall survival rate at 36 months was 97.4% (95% CI: 95-99%). Progression-free survival rate at 3-years was 97.1% (95% CI: 94.9-99.2%), 91.7% (95% CI:84.5-98.3%) and 72.0% (95% CI: 59-87.9%) in the Eutos Long-Term Survival (ELTS) low-, intermediate-, and high-risk group, respectively (P log rank <0.001). Pediatric CML is characterized by bulky disease (large splenomegaly, high leukocyte counts) and the ELTS score can identify children with the poorest outcome.

Authors with CRIS profile

Involved external institutions

Motol University Hospital / Fakultní nemocnice v Motole Czech Republic (CZ) Hospital Boldrini / Centro Infantil Boldrini Brazil (BR) “Aghia Sophia” Children’s Hospital / Νοσοκομείο Παίδων "Η Αγία Σοφία" Greece (GR) St. Anna Kinderkrebsforschung Austria (AT) Rigshospitalet Denmark (DK) Belarusian Research Center for Pediatric Oncology, Hematology and Immunology Belarus (BY) Centre hospitalier universitaire de Poitiers (CHU de Poitiers) France (FR) Prince of Wales Hospital / 威爾斯親王醫院 China (CN) National Institute of Children’s Diseases (NÚDCH) Slovakia (SK) Wrocław Medical University / Uniwersytet Medyczny we Wrocławiu Poland (PL) Children's Hospital Zagreb / Klinika za dječje bolesti Zagreb Croatia (HR) University Hospital Ghent Belgium (BE) Lebanese American University Medical Center - Rizk Hospital (LAUMC-RH) Lebanon (LB) Hôpital Universitaire Robert-Debré France (FR) Technische Universität Dresden Germany (DE) Princess Máxima Center Netherlands (NL)

How to cite

APA:

Millot, F., De Keizer, J., Metzler, M., Kalwak, K., Günes, A.M., De Moerloose, B.,... Suttorp, M. (2026). Children with chronic-phase chronic myeloid leukemia: characteristics and outcomes from the International Registry of Childhood CML. Haematologica, 111(4), 1280-1288. https://doi.org/10.3324/haematol.2025.288283

MLA:

Millot, Frédéric, et al. "Children with chronic-phase chronic myeloid leukemia: characteristics and outcomes from the International Registry of Childhood CML." Haematologica 111.4 (2026): 1280-1288.

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