Changes in the Outcome of High-Risk Pediatric Patients with Acute Lymphoblastic Leukemia, from 2016 to 2024—Single Center, Real-Life Experience

Background and Objectives: Due to 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 one center in Romania, treated with two adapted BFM protocols. Materials and Methods: Patients enrolled were diagnosed with ALL in our center from 01.2016 to 12.2022 and followed up until 12.2024. They were treated according to ALL IC BFM 2009 protocol until 06.2019 and based on ALL AIEOP BFM 2017 protocol since 07.2019. Prognostic factors were analyzed in both subgroups and 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: Comparing the two subgroups, every parameter has 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%). While for standard-risk group events such as relapse or death were isolated, and for intermediate-risk patients they were limited to a small number and did not influence meaningfully the overall outcomes, for high-risk children the results improved noteworthy between the two subgroups. Patients with BCR::ABL1, teenagers, T-ALL had worse outcomes compared to ETV6::RUNX1, smaller children and B-ALL, 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 diagnostic, genetic characteristics and response to treatment, especially prednisone sensibility.