Patient- and caregiver-reported priorities to inform clinical care, research, and therapeutic development
Overview
Craniopharyngioma (CP) is a histologically benign brain tumor; however, treatment often results in significant, lifelong morbidity affecting multiple physiological and neurobehavioral systems. While these sequelae are well documented in the clinical literature, less is known about how patients and caregivers prioritize these outcomes, particularly in relation to daily functioning and long-term life goals.
This study was designed to address that gap by systematically capturing and analyzing patient- and caregiver-reported priorities to inform clinical care, research, and therapeutic development.
Access Full PublicationStudy Rationale
Traditional clinical and research frameworks in CP have focused on tumor control and endocrine outcomes. There is increasing recognition among regulators, researchers, and patient advocacy organizations that patient-reported outcomes are essential to understanding disease burden and guiding innovation.
The objective of this study was to:
- Identify the health challenges most impactful to survivors and caregivers
- Assess how these priorities align with frequency of occurrence
- Define treatment targets that reflect real-world patient needs
Methods
Data were collected through the Hypothalamic–Pituitary Brain Tumors Patient Registry, launched by the Raymond A. Wood Foundation in May 2024.
- Sample size: 161 participants
- Adult CP survivors
- Caregivers, primarily parents of pediatric patients
- Data collected:
- Reported health challenges
- Perceived impact on daily functioning and long-term outcomes
- Prioritization of targets for future treatment development
This registry-based approach enabled the integration of real-world lived experience data into a structured research framework.
Key Findings
Multisystem Disease Burden
Participants identified 17 primary health challenges associated with CP and its treatment, spanning hypothalamic dysfunction, neurological impairments, and visual deficits.
Hypothalamic dysfunction included challenges such as obesity, hyperphagia, sleep dysregulation, thermoregulation, and cognitive and behavioral changes. These findings reinforce the complex, multisystem nature of CP survivorship.
Impact Does Not Correlate with Prevalenc
A central finding of the study is that the most impactful health challenges are not necessarily the most prevalent.
While many symptoms were commonly reported, a subset of 17 challenges emerged as consistently high priority based on their impact on daily life and functional outcomes rather than frequency alone.
Significant Effects on Daily Function and Life Trajectory
The majority of prioritized health challenges were reported to significantly affect activities of daily living, independence, and participation.
Some challenges, particularly changes in social behavior, were also identified as barriers to achieving long-term life goals. These findings highlight areas that are often underrepresented in traditional clinical endpoints.
High Concordance Between Survivors and Caregivers
There was strong overall agreement between adult survivors and caregivers regarding priority health challenges.
Key distinctions included:
- Adult survivors reported greater impact from polydipsia, fatigue, and sleep disturbances
- Caregivers emphasized emotional lability, cognitive challenges, and obesity-related concerns
This alignment strengthens confidence in the consistency of reported priorities across the CP population.
Implications for Therapeutic Development
Nearly all identified health challenges, with the exception of thermoregulation issues, were prioritized as targets for future treatment development.
Specific areas of emphasis included:
- Fatigue and excessive daytime sleepiness
- Obesity and hypothalamic dysfunction
- Cognitive and behavioral impairments
These findings provide clear, patient-informed direction for drug development and clinical research.
Research and Regulatory Significance
The integration of patient and caregiver perspectives into research and therapeutic development is increasingly prioritized by the U.S. Food and Drug Administration and other regulatory bodies.
The results of this study will help inform clinical trial design, improve care delivery, and guide decision-making among researchers, regulators, and payers. Incorporating patient-reported priorities ensures that emerging treatments address outcomes that are meaningful to those living with CP.
Role of the Patient Registry
This study was made possible through participation in the Hypothalamic–Pituitary Brain Tumors Patient Registry.
As a research infrastructure, the registry enables:
- Longitudinal data collection
- Real-world evidence generation
- Identification of unmet clinical needs
Participation in the registry contributes to a growing evidence base that supports improved understanding of CP and accelerates research progress.
Join the RegistryConclusion
This study demonstrates that CP survivorship is characterized by complex, high-burden, multisystem morbidity. It also highlights the importance of integrating patient and caregiver perspectives into research and care.
Aligning clinical and research priorities with lived experience is essential to improving outcomes and advancing the development of meaningful therapies.
Access the Full Publication
Treatment Priorities in Craniopharyngioma: Perspectives of Survivors and Caregivers
https://www.mdpi.com/2227-9059/14/3/664
Advancing the Research
Ongoing participation in the patient registry remains essential to expanding the evidence base, refining research priorities, and accelerating the development of targeted therapies.
Continued contribution of patient-reported data will play a central role in improving outcomes for individuals living with craniopharyngioma.
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