Premature treatment cessation in pediatric acute myeloid leukemia due to excessive toxicity: clinical experiences and patient outcomes - a case series.

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.