To determine whether the population level outcomes of pediatric acute promyelocytic leukemia have improved over time.

We conducted a retrospective analysis of the Surveillance Epidemiology and End Results database for patients with acute promyelocytic leukemia, up to 20 years of age, diagnosed between 1976 and 2016 and actively followed. Patients were stratified based on their period of diagnosis (1976-1989, 1990-1999, 2000-2009, 2010-2016) to assess the temporal trends in overall survival and early mortality.

A total of 553 patients with a median age of 15 years (range, 0-20 years) were included. The 5-year overall survival increased significantly over time (by 22.6% from 1976 to 1989; by 59.2% from 1990 to 1999; by 77.7% from 2000 to 2009; and by 88.9% from 2010 to 2016; P < .001). Early mortality showed an improvement over time in the most recent cohort (by 14% from 1976 to 1989; by 13.5% from1990 to 1999; by 13.3% 2000 to 2009; and by 7.2% from 2010 to 2016) after adjusting for other demographic characteristics in a logistic regression model. On multivariate analysis of overall survival, diagnosis in the earlier time periods was associated with higher mortality as compared with the 2010-2016 period. Age, sex, and race/ethnicity were not significant predictors of overall survival.

Outcomes of pediatric acute promyelocytic leukemia have continued to improve over time at the population level.