This trial is evaluating whether Treatment will improve 2 primary outcomes, 4 secondary outcomes, and 1 other outcome in patients with Watson Syndrome. Measurement will happen over the course of 182 days.
This trial requires 168 total participants across 3 different treatment groups
This trial involves 3 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.
"Data from a recent study from this study suggest that AFNB is a rare disease in the U.S. population, especially among children and young adults. More data are needed to confirm these findings before making public health recommendations." - Anonymous Online Contributor
"The overall 5-year survival rate for patients with neurofibromas was 96% at our institution. The 5-year survival rate for patients with dysembryomas (a benign subtype of neurofibromas) was 98%. Survival was not statistically different between the two groups. Patients with neurofibromas need to be aware of their own risks and may benefit from being able to speak with an expert on this subject." - Anonymous Online Contributor
"The average age of diagnosis of neurofibromas among children was 11.5 yr, and patients were more likely to be male than female. We found no significant differences in terms of the site of occurrence of neurofibromas between Caucasian patients with neurofibromas and non-Caucasian patients with neurofibromas." - Anonymous Online Contributor
"Neurofibromas are rare tumors in children and adolescents. The incidence is higher in black children than in white children and correlates with race-specific genetic alterations in the NF1 gene. The overall incidence of malignant neurofibromas in adults has been estimated at 0.2%. A prospective cohort study is needed to determine the risk factors for developing neurofibromas and the natural history of these lesions in the adult population." - Anonymous Online Contributor
"At present, there are only four drugs approved for neurofibroma as part of an FDA-approved drug label. The Src family kinase inhibitor dasatinib has shown efficacy in some randomized controlled trials with neurofibroma, but this therapy must still be evaluated in larger studies. Until then, we recommend continuing to follow the current standards of care." - Anonymous Online Contributor
"The theoretical basis behind chemotherapy is that the drug interferes with cell growth. This is substantiated by the fact that many different drugs have been demonstrated to kill tumor cells. Chemotherapy is effective because it destroys rapidly dividing tumor cells as well as stromal tumor cells (areas around the tumor where tumor cells grow) as they divide. In addition, chemotherapy kills cancer stem cells, the cells that cause tumors to form. While some treatments do not target cancer stem cells very effectively, others such as gemcitabine are very specific against them. Gemcitabine's effectiveness seems to depend on protein kinase inhibitors, which were developed specifically to block cancer stem cells." - Anonymous Online Contributor
"A review of the literature revealed no cure for neurofibromas. The need for a prospective study was indicated. The aim of this study would be to develop a setup for a multicenter prospective study to assess the effect of various treatments on neurofibroma." - Anonymous Online Contributor
"The most common surgeries performed for patients with neurofibromas are resection and removal of tumors (85%). Facial nerve blocks are also utilized frequently (49%) for pain relief after surgery or radiation treatment of facial nerves. In terms of complications that occur following surgery or radiation, wound dehiscence is the most prevalent (11%), followed by hematoma formation (4%), dysphagia (4%), and pneumothorax (3%). In the event of postoperative complications, surgical intervention can be undertaken at any time if needed. Patients should not hesitate to seek medical advice immediately." - Anonymous Online Contributor
"It was concluded that surgical removal of neurofibromas has been shown to result in long-term benefit in patients' quality of life and cosmetic outcome, but not in overall survival. Considering the risks associated with surgery, chemotherapy appears to be an effective treatment modality in combination with surgeries." - Anonymous Online Contributor
"Treatment options for neurofibromas include surgery (debridement), radiation therapy, and chemotherapy. There is limited evidence to support the use of topical agents such as interferons, imiquimod, and retinoids, although these treatments may slightly improve pain and function. In addition, there is little evidence to support the use of oral agents such as bisphosphonates, teriparatide, or strontium ranelate. The current most common treatment for neurofibromas is surgical debridement, which reduces swelling around the tumor. Some patients do experience recurrence of symptoms after surgery; therefore, for some patients, additional treatment may be needed." - Anonymous Online Contributor
"The cause of neurofibroma remains unknown. It may be related to a genetic mutation though more research is needed. The majority of cases are diagnosed before the age of 15. This suggests that the tumor develops in childhood but it is unclear whether this occurs at birth or if there is a latent period.\n" - Anonymous Online Contributor