Introduction

Hypothalamic dysfunction (HD) during childhood can occur due to (treatment of) a suprasellar brain tumor. HD may not only result in pituitary dysfunction, but also in severe hyperphagia, decreased energy expenditure, disturbed circadian rhythm, temperature dysregulation and behavioral problems. Currently there is no effective treatment for HD. To provide optimal care and to design the most relevant studies, it is essential to understand the patient needs. For this reason, a questionnaire was conducted, to address the most relevant problems and the unmet needs of patients with HD as well as their perspective on future research and clinical approaches.

Methods

Through the different patient advocacy groups, SIOPe craniopharyngioma working group and the ENDO-ERN platform, a world-wide online survey was distributed to patients with childhood onset HD following a brain tumor (April 2022 – October 2022).

Results

In total, 361 patients responded to the survey. Most of the patients had been diagnosed with a craniopharyngioma (78%). In total 63% of the patients had panhypopituitarism. Obesity and fatigue were considered the most important health problems for the patients (in respectively 48.8% and 46.5%). Patients indicated to want more support with food choices, exercise and psychosocial assistance. In total 18% of patients indicated that there is a lack of general information by their doctor. There is a need for more centralized care. Patient ideas for future research included (1) alternative ways for hormone administration (without injection, by pump), (2) new treatments for hypothalamic obesity, (3) ways to improve early diagnosis of suprasellar tumors.

Conclusion

According to the patient perspective, the care of patients with acquired HD should be more centralized, and from an early stage onwards there should be more focus on quality of life and late consequences. Research should focus on fatigue, hypothalamic obesity and early diagnostics. Ideally, care should be delivered by doctors who have a holistic view of the patient in a multidisciplinary expert team.