Although survival in pediatric acute myeloid leukemia (AML) has improved over the past decades, intensive chemotherapy may be associated with severe and life-threatening toxicities. This case series describes pediatric AML patients in whom therapy was discontinued due to excessive toxicity. Patients were identified through a national database of two consecutive AML treatment protocols in the Netherlands (2010-2023). Seven patients were included; discontinuation of treatment was because of a severe fungal infection in five out of seven cases. Five out of seven patients had a favorable genetic profile; four harboring RUNX1::RUNX1T1 and one with a CEBPA-bZIP mutation. All seven patients remain in continuous complete remission with a median follow-up of 6,7 years. This case series shows that selected children with AML who discontinue chemotherapy early due to treatment-related toxicity may still achieve long-term survival and possibly cure. The high incidence of invasive fungal infections highlights a critical cause of treatment-limiting toxicity. This series could inform clinical practice in future similar cases.
This study aimed to identify sociodemographic, clinical, and care-related factors associated with early death in childhood and adolescent cancer patients in Brazil. A retrospective cohort study was conducted using data from the Brazilian Hospital Cancer Registry Integrator Database (IntegradorRHC). All cancer cases diagnosed in individuals aged 0-19 years between 2000 and 2023 were included. Early death was defined as death at the end of the first treatment or within 12 months after diagnosis. Associations were assessed using multivariable logistic regression, and results were expressed as adjusted odds ratios (aOR) with 95% confidence intervals (CI). Among 102,430 eligible cases, early death occurred in 24.7%. Increased risk of early death was independently associated with older age at diagnosis (aOR = 1.006), non-white race/skin color (aOR = 1.467), missing information on race/skin color (aOR = 3.177), unknown referral origin (aOR = 3.507), residence in the Southeast region (aOR = 1.921), and receipt of chemotherapy (aOR = 1.624). Central nervous system tumors (aOR = 2.430), soft tissue sarcomas, bone tumors, and leukemias showed the highest risk of early death. Surgery (aOR = 0.636) and radiotherapy (aOR = 0.750) were associated with reduced risk, while lymphomas (aOR =0.70) showed a protective association. Early death among pediatric cancer patients in Brazil is strongly influenced by tumor type and marked social, regional, and informational disparities.
