Creating a comprehensive research strategy for cutaneous neurofibromas
| Autor: | James Lee, Brigitte C. Widemann, Pierre Wolkenstein, Sharad K. Verma, Jaishri O. Blakeley, Lu Q. Le, Marigo Stathis, Rhonda Jackson |
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| Rok vydání: | 2018 |
| Předmět: |
0301 basic medicine
Research design Request for Applications Clinical Trials as Topic Government geography Medical education Neurofibroma Neurofibromatosis 1 Skin Neoplasms Summit geography.geographical_feature_category Adult patients MEDLINE Cutaneous neurofibroma Dermatology 03 medical and health sciences 030104 developmental biology 0302 clinical medicine Neurology Research Design Humans Neurology (clinical) 030217 neurology & neurosurgery |
| Zdroj: | Neurology. 91:S1-S4 |
| ISSN: | 1526-632X 0028-3878 |
| Popis: | ObjectiveOutside of procedural-based methods, there are currently no established medical treatments for cutaneous neurofibroma (cNF), which afflict up to 99% of patients with NF1. Further, adult patients often report cNF are the greatest burden of living with NF1. The Neurofibromatosis Therapeutic Acceleration Program (NTAP) launched a think tank to address core questions to facilitate development of effective therapeutics for cNF in people with NF1.MethodsExperts (with and without explicit experience with NF1 or cNF) from multiple scientific and medical disciplines, representing the ranks of academia, industry, and government agencies, were invited to become a member of a team addressing a specific subset of questions pertinent to cNF. Teams met monthly to review published and unpublished materials, and created summaries about the material known and unknown that may influence therapeutic development for cNF. Teams prioritized questions and organized supporting data, which was presented to the entire body of experts by each team at a research summit.ResultsFour themes were identified as being relevant to creating a comprehensive research strategy for cNF: (1) establishing definitions of cNF, (2) determining the biology of cNF with respect to tumor initiation, progression, and maintenance, (3) outlining the factors that guide therapies development, and (4) defining core considerations for clinical trials design and optimization for cNF.ConclusionConsiderations and key questions for each of the thematic areas were identified and provided basis for a request for applications launched by NTAP focused on cNF and are described in the accompanying articles of this supplement. |
| Databáze: | OpenAIRE |
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