Pediatric blood & cancer · 2026
Outcomes of Myeloid Leukemia Associated With Down Syndrome Treated With a Reduced-Intensity Protocol: A Multicenter Experience in Argentina.
Клиническое исследование III фазыСтатьяМногоцентровое исследованиеРандомизированное исследованиеНазвание и аннотация: Перевод в очереди
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Deana Alejandra, Gómez Sergio Miguel, Morán Lorena Elizabeth, Felice Maria S, Cecilia Riccheri, Luciana Ferraro Cristina, Gabriela Martinez, Wanda Goldman, Carlos Hollmann, Guillermo Arbesú Mario, Claudia Ruiz, Constanza Drozdowski, Mercedes Tomasetti, Federico Manni, Elizabeth Arrieta Maria, Alejandra Deu, Roberto Navarro Guitart, Horacio Caferri, Gimena Gil María, Brenda Kaltenbach, Luciana Noroña, Leonor Makiya Monica, Lillian Sung
Аннотация
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.
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.