Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1.
Liny JohnGurbani SinghEva DombiPamela L WoltersStaci MartinAndrea BaldwinSeth M SteinbergJessica BernsteinPatricia WhitcombDominique C PichardAnne DufekAndy GillespieKara HeiseyMiriam BornhorstMichael J FisherBrian D WeissAeRang KimBrigitte C WidemannAndrea M GrossPublished in: Clinical trials (London, England) (2023)
This study demonstrates that our observer-rated disfigurement severity score for plexiform neurofibroma was feasible, reliable, and documented improvement in disfigurement in participants with plexiform neurofibroma shrinkage. Prospective studies in larger samples are needed to validate this scale further.
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