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A consistent set of measurement techniques must be applied to reliably and reproducibly evaluate the efficacy of treatments for cutaneous neurofibromas (cNFs) in people with neurofibromatosis type 1 (NF1). cNFs are neurocutaneous tumors that are the most common tumor in people with NF1 and represent an area of unmet clinical need. This review presents the available data regarding approaches in use or development to identify, measure, and track cNFs, including calipers, digital imaging, and high-frequency ultrasound sonography. We also describe emerging technologies such as spatial frequency domain imaging and the application of imaging modalities such as optical coherence tomography that may enable the detection of early cNFs and prevention of tumor-associated morbidity.

Background: As exposure to UV radiation is the primary modifiable environmental risk factor associated with skin cancer, it remains the principal focus of most prevention strategies. Numerous sun protection campaigns have been implemented worldwide; however, their impact on the actual incidence and mortality rates of skin cancer seems to be limited. To create successful skin cancer prevention campaigns, it is important to have a comprehensive understanding of individuals' attitudes and behaviours regarding sun protection. The aim of the current study was to determine and report on the prevalence of self‐reported attitudes, knowledge and behaviours regarding two of the major sun protection recommendations—avoidance of sun exposure and use of sunscreens—in an international representative sample across five continents. Methods: This cross‐sectional study was conducted in 20 countries using a web‐based online survey. Findings: A total of 50,552 individuals, comprising 25,388 men (50.22%) and 25,164 women (49.78%), participated in the survey. Among them, 83.2% reported having been voluntarily exposed to the sun (for sun‐basking reasons) at least once in the last 12 months, and 47.96% acknowledged being exposed to the sun between the hours of 10 AM and 4 PM. The primary reason for non‐adherence was that these hours were the most convenient times (32.28%). Only 24.05% reported applying sunscreen every 2 h when outdoors. Forgetfulness was the primary reason as provided by 27.79% of participants. Males and older age groups were less likely to adopt sun‐protective behaviours around the world. Forgetfulness and the challenges posed by time constraints seem to be the biggest barriers to proper adherence. Interpretation: These findings should prompt the collaboration with health authorities and the manufacturers to enhance adherence by setting reasonable sunscreen prices and creating formulations that make their application less burdensome. [ABSTRACT FROM AUTHOR]

Background: Cutaneous neurofibromas (cNFs) are a major cause of disfigurement in patients with Neurofibromatosis Type 1 (NF1). However, clinical trials investigating cNF treatments lack standardised outcome measures to objectively evaluate changes in cNF size and appearance. 3D imaging has been proposed as an objective standardised outcome measure however various systems exist with different features that affect useability in clinical settings. The aim of this study was to compare the accuracy, precision, feasibility, reliability and accessibility of three imaging systems. Materials and methods: We compared the Vectra‐H1, LifeViz‐Micro and Cherry‐Imaging systems. A total of 58 cNFs from 13 participants with NF1 were selected for imaging and analysis. The primary endpoint was accuracy as measured by comparison of measurements between imaging systems. Secondary endpoints included reliability between two operators, precision as measured with the average coefficient of variation, feasibility as determined by time to capture and analyse an image and accessibility as determined by cost. Results: There was no significant difference in accuracy between the three devices for length or surface area measurements (p > 0.05), and reliability and precision were similar. Volume measurements demonstrated the most variability compared to other measurements; LifeViz‐Micro demonstrated the least measurement variability for surface area and image capture and analysis were fastest with LifeViz‐Micro. LifeViz‐Micro was better for imaging smaller number of cNFs (1–3), Vectra‐H1 better for larger areas and Cherry for uneven surfaces. Conclusions: All systems demonstrated excellent reliability but possess distinct advantages and limitations. Surface area is the most consistent and reliable parameter for measuring cNF size in clinical trials. [ABSTRACT FROM AUTHOR]

Background Cutaneous neurofibromas (cNF) are considered one of the highest burdens of neurofibromatosis type 1 (NF1). To date, no medical treatment can cure cNF or prevent their development. In that context, there is an urgent need to prepare and standardize the methodology of future trials targeting cNF. Objectives The objective was to develop a core outcome domain set suitable for all clinical trials targeting NF1-associated cNF. Methods The validated approach of this work consisted of a three-phase methodology: (i) generating the domains [systematic literature review (SLR) and qualitative studies]; (ii) agreeing (three-round international e-Delphi consensus process and working groups); and (iii) voting. Results (i) The SLR and the qualitative studies (three types of focus groups and a French e-survey with 234 participants) resulted in a preliminary list of 31 candidate items and their corresponding definitions. (ii) A total of 229 individuals from 29 countries participated in the first round of the e-Delphi process: 71 patients, relatives or representatives (31.0%), 130 healthcare professionals (HCPs, 56.8%) and 28 researchers, representatives of a drug regulatory authority, industry or pharmaceutical company representatives or journal editors (12.2%). The overall participation rate was 74%. After round 2, five candidate items were excluded. Between rounds 2 and 3, international workshops were held to better understand the disagreements among stakeholders. This phase led to the identification of 19 items as outcome subdomains. (iii) The items were fused to create four outcome domains ('clinical assessment', 'daily life impact', 'patient satisfaction' and 'perception of health') and prioritized. The seven items that did not reach consensus were marked for the research agenda. The final core outcome domain set reached 100% of the votes of the steering committee members. Conclusions Although numerous outcomes can be explored in studies related to cNF in NF1, the present study offers four outcome domains that should be reported in all trial studies, agreed on by international patients, relatives and representatives of patients; HCPs; 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. [ABSTRACT FROM AUTHOR]

Background: Childhood exposure to ultraviolet radiation (UVR) plays an important role in the development of keratinocyte carcinomas and melanomas. Therefore, sun protective measures ought to be implemented during early childhood. Young children are largely dependent upon adult care providers in order to achieve proper sun protection. Objectives: To develop effective photoprotection in children, it is necessary to understand caregivers' attitudes and knowledge about UVR exposure. This study aimed to explore the variables associated with sun protective behaviour in parents and grandparents during summer vacations. Methods: A multinational, cross-sectional study was conducted using a web-based online survey with a representative sample of parents and grandparents of children aged ≤ 12 years, who cared for their children/grandchildren for at least 2 weeks during the summer of 2021, in five countries (France, Germany, Spain, Italy and the USA). Multiple correspondence analysis (MCA) was used to explore in an unbiased way the possible relationships among all the variables and to identify specific profiles. Results: A total of 6190 adult participants responded to the questionnaire: 5104 parents (average age 42.0 years, 54.3% women) and 1086 grandparents (average age 64.2 years, 55.5% women). MCA allowed discrimination of two groups of respondents based on their answers: a profile with 'unprotected sun exposure habits' vs. those with 'protective sun exposure habits'. Parents fell in closer proximity to the 'unprotected sun exposure habits', and 'sunburn reported in youngest child'. Grandparents adopted more cautious behaviours than parents. Nevertheless, grandparents fell in proximity to 'having exposed the grandchild to the sun between 11 h and 17.00 h', 'not using an umbrella' and 'not using sunglasses'. Conclusions: Although grandparents appear to adopt more cautious behaviours than parents, many gaps in proper sun protection behaviour were observed in both groups of caregivers. Young children are largely dependent upon adult care providers' in achieving proper sun protection. In this study we surveyed 5104 parents and 1086 grandparents regarding their sun protective attitudes and behaviours during the summer vacations. Although grandparents appear to adopt more cautious behaviours than parents, many gaps in proper sun protection behaviour have been highlighted in both caregivers. Important preventive messages to be targeted at grandparents are avoidance of sun exposure when ultraviolet radiation tends to be strongest (11.00–17.00 h), and the use of other strategies for sun protection. Both parents and grandparents might benefit from information regarding proper sunscreen use. [ABSTRACT FROM AUTHOR]

Cutaneous lichen planus is a highly pruritic dermatosis with an unmet need in its management. The aim of this study was to evaluate the short‐term effect and tolerance of high doses of clobetasol propionate 0.05% in cutaneous lichen planus. We conducted a single‐center retrospective cohort study from 2017 to 2021. All adults treated with high‐dose (>5 g/day) clobetasol propionate 0.05% for cutaneous lichen planus were included. Patients with less than 10% affected body surface area at initial presentation or who received concomitant systemic therapy were excluded. The primary endpoint was the rate of complete remission by week 16. Secondary endpoints included maximum daily and median cumulative doses, reduction in pruritus and occurrence of adverse events. Fifty‐seven patients, 60% female, with a mean age of 48 years (min–max,18–83) were included. Cutaneous lichen planus had been present for a median duration of 2 months at initial presentation (min–max, 1–4) and involved a median body surface area of 27%. Pruritus was reported by 55/57 (96%) patients. At week 16, 41/57 (72%) patients had achieved complete remission without treatment modification, among whom 25/41 (61%) had achieved it at week 6. The median daily and cumulative doses were, respectively, 20 g/day (IQR, 10–20) and 560 g (IQR, 320–925). Three patients experienced mild adverse events. No statistical association was demonstrated between the duration of the disease before treatment initiation and clinical response. In conclusion, high‐dose clobetasol propionate 0.05% seems to be an effective, well‐tolerated, and easy‐to‐implement treatment for cutaneous lichen planus. [ABSTRACT FROM AUTHOR]

Background: In 2015, a major achievement in vitiligo research was the development of an internationally agreed upon core outcome domain set for randomized clinical trials (RCTs). Three outcomes were identified as being essential: repigmentation, side-effects/harms and maintenance of gained repigmentation. Four items were further recommended for inclusion. The following recommendations then followed: repigmentation should be assessed by measuring the percentage of repigmentation in quartiles (0–25%, 26–50%, 51–79%, 80–100%) and cosmetic acceptability of the results should be assessed using the Vitiligo Noticeability Scale. Objectives: The primary objective of this study was to assess uptake of the core outcome domain set for RCTs in vitiligo. Secondary objectives were to update the systematic review on outcomes reported in vitiligo RCTs, and to assess whether repigmentation and cosmetic acceptability of the results were measured using the above-mentioned recommended scales. Methods: We searched PubMed, Cochrane Library (CENTRAL and Systematic Reviews) and ClinicalTrials.gov for vitiligo RCTs between November 2009 and March 2021. Screening and data extraction were independently performed on title and summary by two researchers. All outcomes and outcome measures reported in eligible RCTs were retrieved and collated. Results: In total, 174 RCTs were identified: 62 were published between 2009 and 2015, and 112 were published between 2016 and 2021.Thirty-eight different outcomes were reported. Repigmentation was the primary outcome in 89% of trials (150 of 169). Forty-nine different tools were used to measure repigmentation. Side-effects and harms were reported in 78% of trials (136 of 174). Maintenance of gained repigmentation was reported in only 11% of trials (20 of 174) and duration of follow-up varied greatly from 1 to 14 months. Cosmetic acceptability of the results and cessation of disease activity were assessed in only 2% of trials (four of 174). Quality of life of patients with vitiligo was assessed in 13% of trials (22 of 174). Finally, only 11 of 112 RCTs (10%) published between 2016 and 2021 reported all three essential core outcome domains (repigmentation, side-effects and maintenance of gained repigmentation) and none of the trials reported both essential and recommended core outcome domains. Conclusions: Efforts are still needed to close the gap between set recommendations and RCT outcome reporting. [ABSTRACT FROM AUTHOR]

Background: The skin plays an important role in establishing interpersonal relationships, and thus visible skin disorders, which have a significant impact on physical appearance, influence other people's behaviours and attitudes. Objective: To develop and validate a dermatologic‐specific questionnaire to evaluate stigmatization in individuals with visible skin conditions. Methods: Items were generated by a verbatim report based on qualitative interviews with patients with various dermatologic conditions. Subsequently, a study was implemented for psychometric analysis. A dermatology‐specific stigmatization questionnaire (PUSH‐D) was refined via item reduction according to inter‐question correlations, consensus among experts and exploratory factor analysis. Internal consistency was determined by calculating Cronbach's α. Concurrent validity was determined by calculating the correlation between PUSH‐D and the Dermatology Life Quality Index (DLQI) and the Rosenberg Self‐Esteem Scale (RSES). Results: From a primary list of 22 items, PUSH‐D was reduced to a 17‐item questionnaire, covering two pertinent dimensions based on the exploratory factor analysis. Construct validity was demonstrated, and PUSH‐D showed good internal consistency (Cronbach's α = 0.9). PUSH‐D correlated strongly with the DLQI 0.72 (p < 0.001) and moderately with the RSES 0.49 (p < 0.001). Conclusion: PUSH‐D allows a comprehensive view of the degree of stigmatization in visible skin disorders, as well as the comparability of stigmatization levels across various skin conditions. [ABSTRACT FROM AUTHOR]

Introduction and objectives: Inherited epidermolysis bullosa (EB) is a heterogeneous group of genodermatoses characterized by localized or generalized skin and/or mucosal fragility. The objective of this work was to evaluate in France the burden of disease and out‐of‐pocket (OOP) expenditures for families with a child affected by EB. Material and methods: A digital questionnaire was built and distributed to parents of children with EB in partnership with the patients' association DEBRA France. The questionnaire collected clinical and socioeconomic characteristics including the estimated amount of money caregivers had to pay out of their own pockets. The burden of caregivers was assessed using the validated Epidermolysis Bullosa Burden of Disease (EB‐BoD) tool. Linear univariate regression models were conducted to search for factors associated with higher burden and higher OOP. Results: Between October and December 2021, 77 parents answered the questionnaire. The responder was the child's mother in 77% (n = 59) of cases. Parents represented 40 girls and 37 boys with a mean age of 7.5 years and with different EB types and disease severity. The mean BE‐BOD score was 63.9 ± 20.2. The mean score observed in children with severe EB was 69.0 ± 21 versus 59.0 ± 18.6 for moderate/mild. Similarly, the mean BE‐BOD scores observed in parents performing daily wound care were 67.9 ± 19.6. All parents (100%) reported OOP expenses. The mean annual OOP cost was 4129€ ± 4321€. Linear regression demonstrated that for each one‐point increase in the EB‐BoD score, OOP expense increases by 91.1 euros (35.1–147) p = 0.002. Conclusion: EB places a considerable burden on families' daily lives. This burden is closely associated with OOP expenditures to manage EB which were on average 20 times higher compared with the French population. [ABSTRACT FROM AUTHOR]