A core outcome domain set to assess cutaneous neurofibromas related to neurofibromatosis type 1 in clinical trials.

Although numerous outcomes can be explored in studies related to cNF in NF1, the present study offers 4 outcome domains that should be reported in all trial studies, agreed upon by international patients, relatives and representatives of patients, HCP, researchers, representatives of drug regulatory authorities or pharmaceutical companies, and journal editors. The next step will include the development of a set of core outcome measurement instruments to further standardize how these outcomes should be assessed.