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Can prognostic scoring systems for chronic myeloid leukemia as established in adults be applied to pediatric
David Gurrea Salas1, Ingmar Glauche, Josephine T Tauer
1Pediatric Hematology and Oncology, Department of Pediatrics, University Hospital "Carl Gustav Carus", Technical University Dresden, Fetscherstrasse 74, 01307, Dresden, Germany.
Insights
Adult prognostic scores for pediatric chronic myeloid leukemia (CML) show discrepancies. Only the EUTOS score correlated with early treatment response in children, suggesting a need for a tailored pediatric CML risk assessment tool.
Area of Science:
- Hematology
- Pediatric Oncology
- Leukemia Research
Background:
- Specific scoring systems for predicting treatment response in pediatric chronic myeloid leukemia (CML) are lacking, unlike in adult medicine.
- Existing adult prognostic scores (Sokal, Hasford, EUTOS) have not been validated for risk stratification in pediatric CML patients.
Purpose of the Study:
- To evaluate the applicability and comparability of adult prognostic scores (Sokal, Hasford, EUTOS) for risk group categorization in pediatric CML patients.
- To assess the correlation of these adult scores with early treatment response in a pediatric cohort.
Main Methods:
- Analysis of data from 90 pediatric patients in the CML-PAED-II trial treated with imatinib.
- Calculation and application of Sokal, Hasford, and EUTOS scores using patient data.
- Assessment of treatment response at 3 and 6 months based on BCR-ABL1/ABL1 transcript ratios.
Main Results:
- Significant discrepancies were observed when applying adult scores to pediatric patients, with varying percentages categorized into low, intermediate, and high-risk groups.
- Only the EUTOS score demonstrated a significant correlation with early treatment response (transcript ratio >10% at month 3, p=0.008).
- Adjusting the EUTOS score cutoff improved its predictive value for risk categorization in this pediatric cohort.
Conclusions:
- Adult prognostic scores are not directly comparable or fully applicable for risk stratification in pediatric CML.
- The EUTOS score shows potential for predicting early treatment response in children with CML, but requires further validation.
- Development of a pediatric-specific prognostic scoring system for CML is necessary, requiring larger cohorts and longer follow-up.
Abstract:
In contrast to adult medicine, specific scoring systems predicting the treatment response for an individual pediatric patient (pt) with chronic myeloid leukemia (CML) have not yet been defined. We evaluated to what extend prognostic scores as described for adults (e.g., Sokal, Hasford, EUTOS score) resulted in comparable risk group categorizations in a pediatric cohort. Parameters for score calculation were extracted from a data set of 90 patients enrolled into trial CML-PAED-II and treated by a standard dose of imatinib. At month 3 and at month 6, treatment response was analyzed based on the transcript ratio BCR-ABL1/ABL1. By the EUTOS, Hasford, and Sokal scores 81, 59, and 62 % of the patients were categorized as low risk, respectively; 19, 14, and 16 % of the patients as high risk, respectively; and by Hasford and Sokal scores 27 and 22 % of the patients, respectively, as intermediate risk. Twenty-seven out of 72 patients analyzable (38 %) exhibited a transcript ratio >10 % at month 3. We show that only the EUTOS score, but not the Sokal and Hasford score, correlates with this early outcome (p = 0.008). Analyzing the EUTOS score separately, we can demonstrate that lowering the cutoff from 87 to 48 points for categorization in low- and high-risk individuals increases the odds ratio from 2.4 (95 % CI 0.6 to 10.4) to 3.6 (95 % CI 1.3 to 10.9). Data are provided on the distribution of risk categories and resulting discrepancies when adult scores are applied on children and adolescents with CML at diagnosis. A larger number of patients and longer follow-up are still needed to develop a prognostic score specifically adapted to the pediatric and adolescent age cohorts.
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