New data suggests that the current treatment for pediatric adamantinomatous craniopharyngioma (CPA) may not be as effective as it could be.
Current treatment regimens for pediatric CPA are limited to surgery and radiation therapy. This pilot study seeks to identify biologically rational therapeutics for the medical treatment of adamantinomatous CPA by confirming the overexpression of specific molecules.
Study Type
OBSERVATIONAL
Enrollment
250
If available, specimen will be sampled from within the tumor, and not include portions of the tumor capsule. If the tumor includes a cyst, fluid from the cyst will be sampled if available. For each tumor specimen, a companion sample of blood that would otherwise be disposed of through usual clinical practice in the operating room will also be collected.
Children's Hospital Colorado
Aurora, Colorado, United States
Identification of Potential Therapeutic Targets
Investigators will identify potential therapeutic targets through mRNA expression.
Time frame: Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Investigators will identify potential therapeutic targets through microarray analysis.
Time frame: Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Investigators will identify potential therapeutic targets immunohistochemistry.
Time frame: Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Investigators will identify potential therapeutic targets through quantitative PCR.
Time frame: Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Investigators will also perform immunostaining for beta-catenin and use DNA SNaPshot analysis to determine what parts of the tumor are responsible for observed gene signatures.
Time frame: Beginning of study to end of study, up to 1 year.
Survival
The amount of time the patient survives with or without the disease.
Time frame: Beginning of study up to age 21 or death, whichever comes first.
Progression Free Survival (PFS)
The amount of time the patient survives without advancement of disease.
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Time frame: Beginning of study up to age 21 or death, whichever comes first.
Visual Deficit Assessment
With regard to visual deficits, investigators will compare the rates of functional blindness, unilateral blindness, and visual field deficit.
Time frame: At 6 and 12 months after the specimen sample was taken.
Pituitary Function Assessment
With regard to pituitary function, investigators will assess patients based on the quality of life impairment that is associated with their dysfunction. Assessment will be divided among 4 groups: 1. No dependence on hormone supplementation or evidence of diabetes insipidus 2. Dependence on 1 or 2 hormone supplements without diabetes insipidus 3. The presence of diabetes insipidus with or without the need for 2 or fewer hormone supplements 4. Diabetes Insipidus with panhypopituitarism
Time frame: At 6 and 12 months after the specimen sample was taken.