Raymond A. Wood Foundation
Cycle 2
Childhood hypothalamic-pituitary brain tumors can affect the function of the pituitary gland and other nearby structures in the brain. Symptoms include gradual changes in vision, fatigue, excessive urination and headaches. Children with these tumors may grow slowly and may be smaller than expected.
Last updated 04/30/2025
Clinical
Disease Class
Brain tumor
Body Systems
Endocrine
Metabolic
Muscular / Skeletal
Nervous / Sensory
Renal / Urinary / Excretory
Reproductive
Organs
Brain
Known Genetic Link
No, the disease is not known to have a genetic basis
causative_genes
None specified / unknown
contributory_genes
None specified / unknown
Type of Inheritance
Not specified / unknown
Newborn Screening
Not applicable
Disease Mechanism(s)
Unknown
Age of Onset
Adulthood (age 18-64)
Early childhood (age 1+-5)
Middle childhood (6-11)
Average Age at Diagnosis
Adulthood (age 18-64)
Early childhood (age 1+-5)
Middle childhood (6-11)
Life Expectancy
Elderly (age 65+)
Affected Sex(es)
Female
Intersex
Male
National Prevalence
1001-10000
Global Prevalence
10000+
National Incidence
Less than 10
Global Incidence
Less than 10
Populations and/or ancestry with higher prevalence
African-American in the US, certain African countries and Japan
Symptoms / Phenotypes
adipsia / hypodipsia
anxiety
balance problems
behavioral changes
excessive hunger / hyperphagia
growth delay / deficiency
headaches / migraines
intellectual disability
polydipsia
seizures / epilepsy
thermal dysregulation
vision problems
weight gain
Biomarkers
Diagnostic
· Yes for a type pf craniopharyngioma called papillary craniopharyngioma. However, the biomarker development is at the research stage. Lack of funding to validate that biomarker
Prognostic
· Imaging data can help for some prognostic
Therapeutic
· Yes there are some biomarkers to assess therapeutic efficacy for tumor treatments. Other biomarkers, essentially questionnaires serve as biomarkers for efficacy of drugs against hyperphagia. Other anthropometric measures such as weight or BMI for weight loss...
Existing Therapies
FDA-Approved for Symptom Relief
Organizational & Research
Cell Lines
None
Cell Lines, Institution
None
Cell Lines, share
N/A
Disease Model
None
Disease Model, share
N/A
Clinical Trial Role
Focus group
Meeting with regulators
Outcome measures, development
Recruitment and outreach, patients
Recruitment and outreach, trial sites/physicians
Results dissemination, publication
Study material design, review (not protocol)
Study protocol design, review
Biobank, Institution
Not specified
Biobank, Involvement
Funded
Center of Excellence, Institution
None
Registry
Yes, we have a registry that we created
Data Collected, Registry
Imaging data
Longitudinal natural history data
Medication usage
Patient contact info
Patient-reported data
Data Entered by, Registry
Patients
Platform, Registry
IAMRARE
Natural History Study
Yes, we have a natural history study that we created
Data Collected, Natural History Study
Imaging data
Medication usage
Prospective data
Platform, Natural History Study
IAMRARE
FDA Patient Listening Session
Yes
FDA Patient-Focused Drug Development (PFDD) Program
No
ICD Codes
We use an ICD-10 code capturing the family of diseases to which our disease belongs
Diagnostic Guidelines
No
Science Advisory Board Policies
Not Specified
Research Network Policies
Not specified
Research Roadmap
Yes we have a Research Roadmap, and will share policies
International Chapters
None
International Partners
Europe