Understanding Treatment Priorities in Craniopharyngioma

We are pleased to announce the publication of the first study conducted through the Hypothalamic-Pituitary Brain Tumors Patient Registry in Biomedicines. The study entitled “Treatment priorities in Craniopharyngioma: Perspectives of survivors and caregivers” marks an important milestone in advancing patient-centered research in craniopharyngioma (CP).

Using real-world data from 161 registry participants, including both adult survivors and caregivers, the study identifies the health challenges that matter most to those living with CP—offering valuable insight into their impacts on daily lives and long-term outcomes, and define priorities that reflect patient needs.

Participants identified 17 key health challenges, many related to hypothalamic dysfunction, including sleep-wake cycle, fatigue, obesity, hyperphagia, cognitive difficulties, and emotional regulation. Notably, the challenges considered most important were not always the most common, highlighting the need to better align clinical focus with patient experience.

While survivors and caregivers largely agreed on which symptoms had the biggest impact and should be prioritized, some differences did appear. Adult survivors were more impacted by extreme thirst (polydipsia) and place an even greater on developing new treatments for fatigue and excessive daytime sleepiness. Caregivers, on the other hand, highlighted emotional lability and cognitive challenges as the two most impactful symptoms, and placed an even greater importance on developing new treatment for obesity.

As the first publication from the RAWF patient registry, this study represents a meaningful step forward in amplifying patient and caregiver voices in research. 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.

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