Abstract
Abstract
Background
Adamantinomatous craniopharyngioma (ACP) comprises 5-11% of pediatric intracranial tumors and is associated with poor quality of life. In general, cancer outcome studies indicate that females have higher survival rates and better treatment responses than males. Leading theories focus on sex-related genetic/molecular differences versus sex hormone differences as primary contributors to the different outcomes. Here, we investigated sex-associated differences in survival and functional outcomes of prepubescent patients with ACP, aiming to study the role of biological sex in the absence of hormonal effects.
Methods
Sixty-four patients diagnosed with ACP under 7 years of age were retrospectively identified from the Advancing Treatment for Pediatric Craniopharyngioma (ATPC) and the Children’s Hospital Colorado databases. A Cox regression model was used to evaluate time to recurrence/progression. A linear regression model was used to evaluate functional outcomes compared to pre-treatment baseline using a modified Craniopharyngioma Clinical Status Scale (CCSS). Patient age and length of follow-up were included as covariates.
Results
We found that sex is a statistically significant predictor of disease progression (p = 0.0183, power = 0.999), with males demonstrating a 57.4% lower risk of progression (95% CI [13.5%, 79.0%]). In contrast, patient age and follow-up duration did not significantly affect the hazard of progression. There was no statistically significant difference in neurological, visual, pituitary, or hypothalamic functional outcomes between males and females.
Conclusions
Our study finds that in prepubescent ACP patients, males have a lower risk of disease progression than females, although the functional outcomes are equivalent. With an underdeveloped hypothalamic-pituitary-gonadal axis in prepubertal patients, the observed sex difference is likely attributed to factors such as genetic and molecular differences other than hormonal effects. This study highlights the need to identify variables impacting ACP disease course and advocates for personalized treatment strategies to improve patient outcomes and quality of life.