One potential source of new antibacterials is through probing existing chemical libraries for copper-dependent inhibitors (CDIs), i.e., molecules with antibiotic activity only in the presence of copper.
Objective:To assess the perspectives of adults with neurofibromatosis 1 (NF1) regarding cutaneous neurofibroma (cNF) morbidity, treatment options, and acceptable risks-benefits to facilitate the design of patient-centered clinical trials.Methods:An online survey developed by multi-disciplinary experts and patient representatives of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) cNF working group. The survey was distributed to adults with NF1 (n=3734) in the largest international database of individuals with any form of NF. Eligibility criteria included: self-reported NF1 diagnosis, ≥18 years, ≥1 cNF, and ability to read English.Results:548 adults with NF1 responded to the survey. Respondents ranked appearance, number, and then location as the most bothersome features of raised cNFs. Seventy-five percent of respondents considered a partial decrease of 33-66% in the number or size of cNFs as a meaningful response to experimental treatments. Most respondents (48-58%) were willing to try available cNF treatments but were not aware of options outside of surgical removal. Regarding experimental agents, respondents favored topical then oral medications. Most individuals (>65%) reported being “very much” or “extremely willing” to try experimental treatments, especially those with the highest cNF burden. Many respondents were not willing to tolerate side effects like nausea/vomiting (51%) and rash (46%). The greatest barriers to participation in cNF clinical trials were cost of participation and time off work.Conclusions:Most adults with NF1 are willing to consider experimental therapies for treatment of cNFs. These data will guide the design of patient-centered clinical trials for adults with cNFs.
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