Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials
Merker V, Thompson H, Wolters P, Buono F, Hingtgen C, Rosser T, Barton B, Barnett C, Smith T, Haberkamp D, McManus M, Baldwin A, Moss I, Röhl C, Martin S. Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials. Clinical Trials 2023, 21: 6-17. PMID: 38140900, PMCID: PMC10922038, DOI: 10.1177/17407745231205577.Peer-Reviewed Original ResearchPatient-reported outcome measuresClinical trialsOutcome measuresNeurofibromatosis 1Cutaneous neurofibromasDistress ScaleOlder adultsFinal consensus recommendationsKey secondary outcomesPatient-reported outcomesPatient-reported changesAppearance concernsSystematic literature searchNontumor manifestationsTreatment sequelaeSecondary outcomesBACKGROUND/Consensus recommendationsPatient's perspectiveTumor volumePatient representativesGood psychometric propertiesOutcome ratingsLiterature searchPlexiform