This trial is evaluating whether Treatment will improve 1 primary outcome and 2 secondary outcomes in patients with Craniopharyngioma. Measurement will happen over the course of 5 years.
This trial requires 112 total participants across 1 different treatment groups
This trial involves a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
Craniopharyngiomas are not inherited (acquired). They tend to occur in younger children (5-10 years) and are typically nonfunctioning tumors. Craniopharyngiomas affect 1 per 40,000 children, but may not follow a familial pattern. They tend to occur in one or both of the pituitary glands, and are often diagnosed during adolescence. They are diagnosed during one of the first two decades of life, and tend to be benign lesions. The majority of cases continue to grow and may eventually develop a tumor. Most surgeons would exclude optic pathway gliomas as possible causes of persistent headaches.
Results from a recent paper suggests a 50-70% cure rate for patients with stage I and II tumours and no cure for stage III-IV tumours. Patients with stage III tumours should be considered to have a 50 to 60% chance of a cure. Results from a recent paper would not recommend a complete 'wait and see' policy for stage III tumours.
We found over 4 million Americans to have diagnosed with this cancer, of whom 863 died during the study period. The incidence of craniopharyngioma appears to be increasing (with respect to both the number of new cases and the total number of cases). As craniopharyngioma is rare, it appears to be the most common form of pituitary tumour.
This review emphasizes the importance of multidisciplinary treatment approaches in the management of this rare tumor entity and includes specific aspects that have not been previously mentioned in other textbooks (particularly in the neurologic literature).
The majority of patients present with a palpable mass and increased thyroid function abnormalities. A lower prevalence of CPP is reported, and CPP-related symptoms may be absent (15%).
No single treatment for craniopharyngioma is entirely effective. Treatment is based on the tumor's location and the extent of tumor and its components. Radiation therapy remains the modality of choice for most tumors found in the hypothalamus and optic chiasm. In the midline, surgery is often an effective treatment for tumors with extracerebral extension or cavernous sinus invasion. Complete surgical resection is not always available, since these tumors can spread to other parts of the brain. Surgical resection should be complemented by stereotactic approaches and/or irradiation, depending on the extent of residual tumor.
About 0.6% of people in the general population develop a craniopharyngioma in their lifetime. About 15% of people with pituitary tumours develop one. However, the probability of developing a craniopharyngioma is higher in patients with tumors that tend to have a less benign growth pattern and in patients who present with symptoms.
We found that tumor dissemination occurred in <24 hours in most patients. This early tumor dissemination warrants further investigation as a marker to detect potential disseminated disease to help tailor multimodality therapy for patients with advanced tumors.
Treatment for CRH and PRH causes a marked improvement in anosmia in patients with CRH tumors and has a longer interval of benefit in patients with PRH tumors. Further studies seem warranted for longer follow-up studies and a randomized study to confirm these results.
Most conventional treatments for craniopharyngiomas have limited efficacy and the majority of patients die by the time a diagnosis is made. Despite this response the survival rate following a craniopharyngioma diagnosis is relatively good. However, treatment has little effect on symptoms and quality of life. This lack of effect, combined with the lack of significant long term survival after therapy means craniopharyngiomas are a challenging and misunderstood tumours.
The authors suggest that aggressive management by experienced neurosurgeons for severe cases is warranted. These patients should have a strong desire to receive the best care possible.
This is the first report of CPH1H as a bona fide autosomal dominant disorder. We have thus demonstrated an autosomal recessive inheritance pattern for craniopharyngioma, the first gene for this disease to be identified, and the first locus identified.