Retrospective dataset and survey analyses identify gaps in data collection for craniopharyngioma and priorities of patients and families affected by the disease

Abstract

Craniopharyngioma is a rare type of brain tumor located in the suprasellar region of the brain, close to important structures like the pituitary gland. This study aimed to better understand patient outcomes associated with existing therapies and to inform new treatment strategies.

Our team collected clinical, genomic, and outcome data from a retrospective cohort of patients with craniopharyngioma through the Children's Brain Tumor Network database. In addition, a survey was conducted among patients and families with a diagnosis of craniopharyngioma to gain insight into their experiences during diagnosis and treatment.

Our reach found that a large number of patients had specimens available for sequencing, but data on quality of life and functional outcomes were lacking. Reported patient comorbidities were also found to be lower than previously reported. The survey results showed that treatment considerations differed at the time of diagnosis compared to recurrence, and patients and families preferred therapies that improved quality of life rather than reducing the risk of recurrence. Endocrine issues were found to have the greatest impact on patients' lives.

Our findings emphasizes the need for prospective collection of quality of life and functional metrics, along with clinical and molecular correlates, inindividuals with craniopharyngioma. This will help to develop more effective treatments that prioritize patients' needs.