Children with chronic-phase chronic myeloid leukemia: characteristics and outcomes from the International Registry of Childhood CML
Year: 2025
Type: article
Study design: Observational Study
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.
Source: Haematologica
Authors: Frédéric Millot, Joe De Keizer, Markus Metzler, Krzysztof Kalwak, Adalet Meral Günes, Barbara De Moerloose, Maaike Luesink, Antonio Molines Honrubia, Petr Sedlacek, Julia Yajima, Mirella Ampatzidou, Michael Dworzak, Birgitte Lausen, Marina Borisevich, Chi Kong Li, Alexandra Kolenova, Gordana Jakovljevic, Roula Farah, Lisa Durocher, André Baruchel, Meinolf Suttorp
Institutions: Inserm, Centre Hospitalier Universitaire de Poitiers, Universitätsklinikum Erlangen, University of Wrocław, Wroclaw Medical University, Ghent University Hospital, Princess Máxima Center, Hospital Universitario Insular de Gran Canaria, Charles University, University Hospital in Motol, Centro Infantil Boldrini, Children's Hospital Agia Sophia, St Anna Children's Hospital, St. Anna Children's Cancer Research Institute, Copenhagen University Hospital, Rigshospitalet, Belarusian Research Center For Pediatric Oncology and Hematology, Prince of Wales Hospital, Children's Hospital Zagreb, University Medical Center Rizk Hospital, Hôpital Robert-Debré, Technische Universität Dresden
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,157
OpenAlex ID: W4415461322