Related Experiment Video
Updated: May 5, 2026
Murine Model of Leukemia Relapse to Induction Chemotherapy for Acute Lymphoblastic Leukemia
Published on: October 17, 2025
Outcomes of Myeloid Leukemia Associated With Down Syndrome Treated With a Reduced-Intensity Protocol: A Multicenter
Alejandra Deana1, Sergio Miguel Gómez1, Lorena Elizabeth Morán1
1Grupo, Argentino de Tratamiento de la Leucemia Aguda (GATLA), Buenos Aires, Argentina.
Insights
Pediatric myeloid leukemia in Down syndrome (ML-DS) patients in Argentina showed 80.3% survival with a reduced-intensity protocol. Treatment-related mortality was 12.5%, indicating a need for improved supportive care.
Area of Science:
- Pediatric Oncology
- Hematology
- Genetics
Background:
- Children with Down syndrome (DS) have a significantly higher risk of myeloid leukemia (ML-DS).
- While survival rates are high in developed countries, treatment toxicity is a major concern in other regions.
- This study focuses on outcomes in pediatric ML-DS patients in Argentina.
Purpose of the Study:
- To evaluate overall survival (OS), event-free survival (EFS), cumulative incidence of relapse (CIR), and treatment-related mortality (TRM).
- To assess the efficacy and safety of a reduced-intensity chemotherapy protocol in pediatric ML-DS patients in Argentina.
Main Methods:
- A multicenter retrospective study of 49 pediatric patients (≤18 years) with ML-DS treated between 2008 and 2025.
- Patients received the GATLA 8-LMAP-2007 protocol, a modified, reduced-intensity regimen.
- Survival outcomes were analyzed using Kaplan-Meier estimates and log-rank tests; cranial irradiation and stem cell transplantation were not used.
Main Results:
- The study included 49 patients, with 59.2% being ≤2 years old and 75.5% presenting with FAB M7 subtype.
- At 48 months, the cumulative incidence of relapse (CIR) was 6.9% and treatment-related mortality (TRM) was 12.5%.
- Both 48-month event-free survival (EFS) and overall survival (OS) were 80.3%.
Conclusions:
- An adapted, reduced-intensity protocol for ML-DS in this Argentine cohort resulted in favorable survival rates.
- The observed TRM of 12.5% underscores the critical need for enhanced supportive care strategies.
- Further refinement of treatment protocols is necessary to minimize treatment-related toxicity in pediatric ML-DS patients.
Background:
Children with Down syndrome (DS) have a markedly increased risk of myeloid leukemia (ML-DS). While survival rates approach 90% in high-income settings, treatment-related toxicity remains a critical challenge in other regions. This study evaluated overall survival (OS), event-free survival (EFS), cumulative incidence of relapse (CIR), and treatment-related mortality (TRM) in pediatric patients with ML-DS in Argentina.
Design/Methods:
We conducted a multicenter retrospective study of 49 patients with ML-DS (≤18 years) treated between 2008 and 2025. All patients were treated with the GATLA 8-LMAP-2007 protocol, a reduced-intensity regimen based on AML-BFM 98 (four chemotherapy cycles with reduced anthracyclines, omission of high-dose cytarabine and mitoxantrone, eight intrathecal doses, and 1-year maintenance). We did not use cranial irradiation and hematopoietic stem cell transplantation. Survival was estimated using the Kaplan-Meier method, and comparisons were performed using the log-rank test.
Results:
We enrolled 49 patients with ML-DS in this study. Most patients were ≤2 years old (59.2%) and presented with the FAB M7 subtype (75.5%). At 48 months, the CIR was 6.9% (standard error [SE] 3.9), and TRM was 12.5% (SE 4.8). Both 48-month EFS and OS were 80.3% (SE 5.9).
Conclusions:
In this Argentine cohort, ML-DS treated with an adapted reduced-intensity protocol achieved favorable survival outcomes. However, the TRM of 12.5% highlights the ongoing need to optimize supportive care and further refine strategies to reduce treatment-related toxicity in this population.
More Related Videos
09:57Comprehensive Protocol to Sample and Process Bone Marrow for Measuring Measurable Residual Disease and Leukemic Stem Cells in Acute Myeloid Leukemia
Published on: March 5, 2018
06:39Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
