Although craniopharyngioma (CP) is considered a benign tumor, the aftermath of tumor removal and treatment leaves survivors and their families facing many health challenges. While these issues are well recognized by doctors, less is known about which problems survivors and their families feel are the most important to address.
Thanks to the community’s participation in the Hypothalamic-Pituitary Brain Tumors Patient Registry, launched in May 2024, the research team at the Raymond A. Wood Foundation was able to collect and analyze data from 161 people—both adult CP survivors and caregivers, mostly parents of children with CP. Participants shared their real-life perspectives on the health challenges that matter most to them, especially those affecting daily life and long-term goals, as well as the issues they hope will be the focus of new treatments.
What were the main findings?
The majority of study participants identified 17 main health challenges, including problems associated with dysfunction of the brain’s hypothalamus, neurological issues, and vision impairment. Hypothalamic dysfunction covered issues like trouble controlling body temperature, sleep challenges, obesity, hyperphagia, cognition, behaviors. Interestingly, health challenges that matter most, were not always the ones that happened most often.
These 17 challenges mostly affected survivors’ daily activities, and some—like changes in social behavior—also made it harder for survivors to achieve long-term goals. Both survivors and caregivers mostly agreed on which health problems were most important. The main difference was that adult survivors were more impacted by extreme thirst (polydipsia). For adult survivors, fatigue and sleep problems were among the top four most serious issues impacting daily life. For caregivers, rapid and often unpredictable changes in mood or emotions (emotional lability) and cognitive issues were the most impactful for those they cared for. Nearly all of these health challenges—except problems with temperature control—were chosen as the main priorities for new treatments by both groups.
Adult survivors placed special importance on developing treatments for fatigue and excessive daytime sleepiness, while caregivers were placing a special importance on new obesity treatment.
How does this study make a difference in the lives of the CP community?
Collecting and integrating the perspectives, values, and preferences of patients or their caregivers about their condition in the development of new treatments, has become a key focus for the Food and Drug Administration (FDA), other regulatory agencies, and health insurers.
The results of this study will help doctors provide better care and support for families; while also helping researchers, drug developers, regulators, and payers better understand what matters most to patients, and guide the development of new treatments that truly meet the needs of people living with CP.
For the CP community- how you can help to advance CP research and treatment:
If you have already enrolled in the Hypothalamic Pituitary Tumors Patient Registry, thank you. Your voice was likely a part of what made this study possible. As a CP survivor or caregiver, you have the power to help advance understanding of this rare disease by enrolling in the patient registry.
When you share your health information and lived experience, you contribute valuable data that helps researchers and clinicians better understand the long-term effects of CP, identify patterns in treatment and outcomes, and guide future studies.
Your participation strengthens the knowledge base needed to accelerate scientific discovery, improve medical care, and ultimately enhance the quality of life for you and for future survivors. Learn more and enroll in the registry here.
Read more about the study here.