Effect of Treatment in a Specialized Pediatric Hemato-Oncology Setting on 5-Year Survival in Acute Lymphoblastic Leukemia: A Quasi-Experimental Study

Simple Summary Adolescents and young adults (AYAs) with acute lymphoblastic leukemia (ALL) have a worse prognosis than children. In addition to differences in biology-such as higher incidence of unfavorable genetic alterations in the AYA population-this might be related to the fact that ALL patients under a certain age (often 18 years) are generally treated in special pediatric hemato-oncology settings, which is associated with improved survival, while patients above that age are treated in adult hemato-oncology care settings. Based on previous research, adult treatment settings have increasingly adopted pediatric-inspired protocols, which appear to have led to increased survival of adolescent ALL patients. This study aims to assess whether there remains an effect of treatment of ALL patients in a specialized pediatric hemato-oncology setting on 5-year survival. This study provides insight into the effects of such treatment for ALL patients, and may stimulate further research into causal relationships in other oncological conditions. Survival rates of adolescents and young adults (AYAs) with acute lymphoblastic leukemia (ALL) are inferior to those of pediatric ALL patients. In part, this may be caused by differences in treatment setting. Generally, children are treated in specialized pediatric hemato-oncology settings, whereas AYAs are treated in adult hemato-oncology settings. Since 2005, adult treatment protocols have included pediatric-inspired chemotherapy, which has been the standard of care for AYAs from 2008 onwards. This study aims to assess whether, despite protocols in both settings having become more similar, there remains an effect of treatment in specialized pediatric hemato-oncology settings on 5-year survival for ALL patients in the Netherlands. We used nationwide registry data (2004-2013) on 472 ALL patients aged between 10 and 30 years old. A fuzzy regression discontinuity design was applied to estimate the treatment effect using two-stage least squares regression with the treatment threshold at 17 years and 7 months of age, adjusting for sex, age at diagnosis, and immunophenotype. We found a risk difference of 0.419 (p = 0.092; 95% CI = -0.0686; 0.907), meaning a 41.9 percentage point greater probability of surviving five years after diagnosis for ALL patients treated in specialized pediatric hemato-oncology settings. Our results suggest that ALL patients around the threshold could benefit from increased collaboration between pediatric and adult hemato-oncology in terms of survival.