Endocrine sequelae after pediatric craniopharyngioma treatment: a single-center retrospective cohort study

Abstract

Abstract Introduction: Craniopharyngiomas (CP) are rare brain tumors with a low mortality rate, but with significant morbidity, in part due to the various long-term endocrine sequelae related to hypothalamic/pituitary deficiencies. Our objective was to assess the prevalence of endocrine dysfunction and outcome after treatment of CP at our institution and to apply the novel diagnostic criteria for hypothalamic syndrome (HS). In addition, we give an overview of treatments already attempted for hypothalamic obesity (HO). Methods This retrospective cohort study included children treated and followed up for CP at the pediatric oncology and endocrinology department at University Hospitals Leuven between January 2000 and December 2020. Clinical and endocrine characteristics were collected during a 5-year period following diagnosis of CP. The Müller radiological criteria and the novel diagnostic criteria for HS were applied. A brief literature review regarding treatments already attempted for HO was conducted. Results Fifteen patients were included and all of them developed endocrine sequalae over time, with 7 patients (47%) experiencing failure of at least one endocrine axis at diagnosis of CP and 7 patients (47%) developing panhypopituitarism. The diagnosis of HO was clinically made in 8 patients (54%). Ten patients (67%) met the diagnostic criteria for HS. Although novel therapeutic approaches provide promising perspectives, no therapy for HO in children has been proven to be effective. Conclusion Long-term pituitary deficiencies and HO after pediatric CP are highly prevalent. Continuing multidisciplinary care to improve the quality of life of these patients is necessary. International cooperation and further long-term prospective trials for the treatment of HO are needed.