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Changes in the Outcome of Pediatric Patients with Acute Lymphoblastic Leukemia-Single Center, Real-Life Experience
Letitia E Radu1,2, Andra D Marcu1,2, Ana M Bica1,2
1Faculty of General Medicine, "Carol Davila" University of Medicine and Pharmacy, 020021 Bucharest, Romania.
Insights
Pediatric acute lymphoblastic leukemia (ALL) survival rates improved significantly in Romania, with overall survival reaching nearly 90% and high-risk patient survival up to 80%. Treatment protocol adaptations led to better outcomes, highlighting the importance of prognostic factors like age and genetics.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Research
Background:
- Pediatric acute lymphoblastic leukemia (ALL) survival rates have improved globally, but disparities persist between high-income and limited-resource settings.
- This study addresses the need for outcome analysis in resource-limited centers using adapted treatment protocols.
Purpose of the Study:
- To analyze prognostic factors and treatment outcomes in a cohort of 223 pediatric ALL patients treated at a single Romanian center.
- To evaluate the impact of two adapted Berlin-Frankfurt-Münster (BFM) protocols on patient survival and disease recurrence.
Main Methods:
- Retrospective analysis of 223 pediatric ALL patients diagnosed between January 2016 and December 2022.
- Patients were treated with either the ALL IC BFM 2009 or the ALL AIEOP BFM 2017 protocol.
- Key outcomes measured included event-free survival (EFS), overall survival (OS), cumulative incidence of relapse (CIR), relapse-free survival (RFS), and non-relapse mortality (NRM).
Main Results:
- Significant improvements were observed with the newer protocol across all measured parameters, including increased complete remission rates and 5-year OS (88.18% vs. 82.54%).
- High-risk pediatric ALL patients showed substantial survival improvements, with rates reaching up to 80% with the adapted protocols.
- Identified negative prognostic factors included older age (teenagers), T-cell phenotype, and the presence of the BCR::ABL1 fusion gene.
Conclusions:
- The adapted BFM protocols led to a notable increase in 5-year OS for pediatric ALL patients, approaching 90% in the analyzed cohort.
- Treatment protocol adaptations and careful consideration of prognostic factors such as age, genetics, and treatment response are crucial for improving outcomes in pediatric ALL, especially in resource-limited settings.
- The study underscores the successful implementation of advanced treatment strategies in a single center, improving survival rates for high-risk pediatric ALL patients.
Abstract:
Background and Objectives: Due to the progress made in all areas of research, pediatric patients diagnosed with acute lymphoblastic leukemia (ALL) now have an average overall survival rate of 90%. There are still discrepancies between high-income countries and limited-resource centers. The aim of this study was to analyze prognostic factors and outcome parameters in a 223-patient cohort from a single center in Romania, treated with two adapted BFM protocols. Materials and Methods: The patients diagnosed with ALL in our center were enrolled in this study from January 2016 to December 2022 and subsequently followed up until December 2024. The patients were treated first according to the ALL IC BFM 2009 protocol until June 2019 and afterwards with the ALL AIEOP BFM 2017 protocol starting with July 2019. The prognostic factors were analyzed in both subgroups and the outcomes were measured: event-free survival (EFS), overall survival (OS), cumulative incidence of relapse (CIR), relapse-free survival (RFS) and non-relapse mortality (NRM). Results: The comparison between the two subgroups revealed that every parameter improved over time: complete remission after induction (87.75% vs. 80.7%), early deaths (3.92% vs. 5.78%), deaths in remission (4.08% vs. 5.26%), 5-year EFS (73.79% vs. 70.22%), 5-year CIR (18.36% vs. 19.04%), 5-year RFS (81.76% vs. 80.97%), 5-year NRM (7.85% vs. 10.77%), and 5-year OS (88.18% vs. 82.54%). Whereas for the standard-risk group, events such as relapse or death were isolated, for intermediate-risk patients, the events were limited to a small number and did not significantly influence the overall results, and for high-risk children, the results improved significantly between the two subgroups. The worst outcomes were observed in patients with the BCR::ABL1 fusion gene, T-cell phenotype, and in teenagers, compared to the ETV6::RUNX1 fusion gene, B precursor ALL, and in smaller children, respectively. Conclusions: The 5-year OS increased in our center from 82.54% to almost 90%, with the most substantial finding being the survival rate for high-risk patients, now reaching up to 80%. The prognostic factors were age at diagnosis, genetic characteristics, and response to treatment, especially prednisone sensibility.
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