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Summary Despite major improvements in the cure rate of childhood acute myeloid leukaemia (AML), 5–15% of patients still die from treatment‐related complications. In a historical prospective cohort study, we analysed the frequency, clinical features and risk factors for early deaths (ED) and treatment‐related deaths (TRD) in 525 children included in the Nordic Society of Paediatric Haematology and Oncology (NOPHO)‐AML‐84, ‐88 and ‐93 trials. Seventy patients (13%) died before starting treatment or from treatment‐related complications. The death rate rose from 11% in NOPHO‐AML‐84 to 29% in ‐88, but then fell to 8% in ‐93. Sixteen patients (3%) died within the first 2 weeks, mainly from bleeding or leucostasis. Hyperleucocytosis, age <2 or ≥10 years were risk factors. After day 15, 10% of patients died from treatment‐related complications with infection as the main cause of death. Risk factors were age <2 or ≥10 years and treatment according to the NOPHO‐AML‐88 protocol. The number of EDs and TRDs in AML is high. Therefore optimal antifungal prophylaxis is essential, and studies on the benefit of antibacterial prophylaxis and individual risk factors for ED and TRD are needed.

The self-reported health of children treated on NOPHO-AML protocols without HSCT was good, and their use of health care services was limited. Reported health and social outcomes were comparable to those of their siblings. Many survivors were smoking which may increase the risk of late effects.