TY - JOUR
T1 - cNF-Skindex in Adults Living with Neurofibromatosis 1
T2 - Severity Strata in France and Validation in United States Adults
AU - Fertitta, Laura
AU - Sarin, Kavita Y.
AU - Bergqvist, Christina
AU - Patel, Ekshika
AU - Peiffer, Bastien
AU - Moryousef, Sabine
AU - Armand, Marie Laure
AU - Jannic, Arnaud
AU - Ferkal, Salah
AU - Ravaud, Philippe
AU - Tran, Viet Thi
AU - Blakeley, Jaishri O.
AU - Romo, Carlos G.
AU - Ezzedine, Khaled
AU - Wolkenstein, Pierre
N1 - Publisher Copyright:
© 2023 The Authors
PY - 2023/11
Y1 - 2023/11
N2 - Cutaneous neurofibromas (cNF) contribute to the impairment of QOL in individuals with neurofibromatosis 1. The cNF-Skindex, validated in a French population, specifically assesses the cNF-related QOL. In this study, we first defined severity strata using an anchoring approach on the basis of patient's burden. In total, 209 patients answered the anchor question and the cNF-Skindex. We tested the agreement among the three strata, generated by all potential couples of cut-off values of the cNF-Skindex and the three strata defined in the anchor question. The cut-off values 12 and 49 provided the highest Kappa value (κ = 0.685, 95% confidence interval = 0.604−0.765). Second, we validated the score and the strata in a United States population using the answers provided by 220 French and 148 United States adults. In the multivariable linear regression analysis, the country of origin was not a factor associated with the score (P = 0.297). The number of cNF along the different severity strata was similar between the French and the United States populations. In conclusion, stratification constitutes a powerful tool to better interpret the cNF-Skindex in daily practice and in clinical trials. This study validates its use in two populations that together constitute a large cohort of patients willing to participate in clinical research.
AB - Cutaneous neurofibromas (cNF) contribute to the impairment of QOL in individuals with neurofibromatosis 1. The cNF-Skindex, validated in a French population, specifically assesses the cNF-related QOL. In this study, we first defined severity strata using an anchoring approach on the basis of patient's burden. In total, 209 patients answered the anchor question and the cNF-Skindex. We tested the agreement among the three strata, generated by all potential couples of cut-off values of the cNF-Skindex and the three strata defined in the anchor question. The cut-off values 12 and 49 provided the highest Kappa value (κ = 0.685, 95% confidence interval = 0.604−0.765). Second, we validated the score and the strata in a United States population using the answers provided by 220 French and 148 United States adults. In the multivariable linear regression analysis, the country of origin was not a factor associated with the score (P = 0.297). The number of cNF along the different severity strata was similar between the French and the United States populations. In conclusion, stratification constitutes a powerful tool to better interpret the cNF-Skindex in daily practice and in clinical trials. This study validates its use in two populations that together constitute a large cohort of patients willing to participate in clinical research.
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U2 - 10.1016/j.jid.2023.04.014
DO - 10.1016/j.jid.2023.04.014
M3 - Article
C2 - 37149083
AN - SCOPUS:85162862353
SN - 0022-202X
VL - 143
SP - 2226-2232.e1
JO - Journal of Investigative Dermatology
JF - Journal of Investigative Dermatology
IS - 11
ER -