Treatment Outcomes and Prognostic Factors in Children with Myeloid Leukemia Associated with Down Syndrome: A Nationwide Polish Retrospective Study

Background: Significant progress has been achieved in the treatment of children with myeloid leukemia associated with Down syndrome (ML-DS). We aimed to evaluate treatment outcomes and prognostic factors in children with ML-DS treated in Poland. Methods: Thirty-six children with ML-DS treated at pediatric hematology and oncology centers in Poland according to the ML-DS 2006 protocol based on the AML-BFM backbone between 2015 and 2024 were retrospectively analyzed. Results: The estimated 5-year event-free survival (EFS) and overall survival (OS) were 84.3% (95%CI: 72.5–98.1%) and 87.9% (77.4–99.8%), respectively. Although children with a complex karyotype showed numerically lower survival estimates than those with isolated constitutional trisomy 21 or additional cytogenetic abnormalities (OS: 60% [95%CI: 29.3–100%] vs. 90% [73.2–100%] vs. 92.3% [78.9–100%], p = 0.24; EFS: 60% [95CI: 29.3–100%) vs. 75% [49.6–100%] vs. 92.3% [78.9–100%], p = 0.26), p = 0.26), these differences were not statistically significant. MRD positivity (≥0.1%) after the second induction cycle was identified as an adverse prognostic factor in the univariable analysis (HR = 31.1, 95% CI: 2.74–353.2; p = 0.006). Conclusions: Persistent MRD positivity after the second induction cycle may identify children with ML-DS at increased risk of treatment failure. Larger multicenter studies are needed to validate the prognostic significance of MRD and recurrent molecular alterations to optimize risk-adapted treatment strategies.

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Journal
Journal of Clinical Medicine
Published
2026-09-28
DOI
https://doi.org/10.3390/jcm15197548
Primary Topic
Acute Myeloid Leukemia Research
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article
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article

Treatment Outcomes and Prognostic Factors in Children with Myeloid Leukemia Associated with Down Syndrome: A Nationwide Polish Retrospective Study

Bartosz Chyżyński, Wojciech Michal Mlynarski, Krzysztof Kałwak, Tomasz Urasiński et al.
Journal of Clinical Medicine
Acute Myeloid Leukemia Research
article

Treatment Outcomes and Prognostic Factors in Children with Myeloid Leukemia Associated with Down Syndrome: A Nationwide Polish Retrospective Study

Bartosz Chyżyński, Wojciech Michal Mlynarski, Krzysztof Kałwak, Tomasz Urasiński, Radosław Chaber, Agnieszka Mizia‐Malarz, Joanna Zawitkowska, Pawel Jozef Laguna, Weronika Stolpa, Szymon Skoczeń, Anna Rodziewicz-Konarska, Monika Lejman, Katarzyna Maria Muszyńska-Rosłan, Katarzyna Pawińska-Wąsikowska, Karolina Bukowska-Straková, Anna Tomoń, Katarzyna Ł. Bobeff, Teofila Książek, Magdalena Samborska, Agnieszka Pac, Jacek Wachowiak, Małgorzata Czogała, Ninela Irga‐Jaworska, Katarzyna Mycko, Wanda Badowska, Jan Styczyński, Paulina Deleszkiewicz, Robert Dębski, Marta Kozłowska, Karolina Zielezińska, Walentyna Balwierz
article en

Abstract

Background: Significant progress has been achieved in the treatment of children with myeloid leukemia associated with Down syndrome (ML-DS). We aimed to evaluate treatment outcomes and prognostic factors in children with ML-DS treated in Poland. Methods: Thirty-six children with ML-DS treated at pediatric hematology and oncology centers in Poland according to the ML-DS 2006 protocol based on the AML-BFM backbone between 2015 and 2024 were retrospectively analyzed. Results: The estimated 5-year event-free survival (EFS) and overall survival (OS) were 84.3% (95%CI: 72.5–98.1%) and 87.9% (77.4–99.8%), respectively. Although children with a complex karyotype showed numerically lower survival estimates than those with isolated constitutional trisomy 21 or additional cytogenetic abnormalities (OS: 60% [95%CI: 29.3–100%] vs. 90% [73.2–100%] vs. 92.3% [78.9–100%], p = 0.24; EFS: 60% [95CI: 29.3–100%) vs. 75% [49.6–100%] vs. 92.3% [78.9–100%], p = 0.26), p = 0.26), these differences were not statistically significant. MRD positivity (≥0.1%) after the second induction cycle was identified as an adverse prognostic factor in the univariable analysis (HR = 31.1, 95% CI: 2.74–353.2; p = 0.006). Conclusions: Persistent MRD positivity after the second induction cycle may identify children with ML-DS at increased risk of treatment failure. Larger multicenter studies are needed to validate the prognostic significance of MRD and recurrent molecular alterations to optimize risk-adapted treatment strategies.

Journal of Clinical MedicineVol. 15(19)
Poznan University of Medical Sciences (PL), Medical University of Lublin (PL), Jagiellonian University (PL), Medical University of Białystok (PL), Medical University of Silesia (PL), Medical University of Warsaw (PL), Nicolaus Copernicus University (PL), Wroclaw Medical University (PL), Uniwersytecki Szpital Dziecięcy (PL), Medical University of Lodz (PL), Collegium Medicum in Bydgoszcz (PL), Gdańsk Medical University (PL), Pomeranian Medical University (PL), University of Rzeszów (PL)
Good health and well-being
Openalex Percentile: Top 11%
Acute Myeloid Leukemia Research
DOIOpen Access PDFOpenAlex
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