British Journal of Dermatology最新文献

Background: Pemphigus is an autoimmune bullous disease (AIBD) and has two main subtypes, pemphigus vulgaris (PV) and pemphigus foliaceus (PF). For adequate interpretation and comparison of clinical studies in pemphigus, it is essential to have outcomes and outcome measurement instruments (OMIs) that are well-defined, uniform and relevant.

Objectives: To provide a comprehensive overview of previously reported outcomes and OMIs in pemphigus clinical research over the past two decades.

Methods: A systematic literature search was performed in the databases MEDLINE, Embase, CINAHL, PsycINFO, Cochrane CENTRAL, Web of Science and trial registries covering the period between January 2002 and December 2023. Clinical trials, prospective cohort studies and systematic reviews were included. Retrospective studies were excluded. All identified outcomes and their respective OMIs were grouped into outcome domains and areas.

Results: From all the included studies (n = 205), a total of 1502 outcomes were extracted verbatim, categorized in 55 outcome domains and 11 outcome areas. We found a gradual increase in the number of published trials over the past two decades. The included studies mainly focused on 'clinical response' (92%), 'safety monitoring' (75%) and 'immune response' (61%). 'Quality of life' was sparsely reported (12%). Heterogeneity of reported outcomes was observed across all study designs. Within the outcome area 'clinical response', the proportion of defined outcomes based on the 2008 consensus statement has not been consistent over time. However, four recent clinical trials used primary endpoints based on the 2008 consensus definitions. Moreover, a broad spectrum of outcome measurement instruments (n = 65) was reported.

Conclusions: This review showed a wide variety in reported outcomes and OMIs in pemphigus clinical research over the past two decades. Despite standardization efforts, most studies still lack well-defined, consistent and relevant outcomes and OMIs. However, a positive trend is emerging as four recent trials did use consensus-based definitions and patient-focused OMIs. Nevertheless, the outcomes are overly stringent, lack patient-centeredness, and do not include intermediate endpoints. Therefore, refinement of the 2008 expert consensus statement through collaborative efforts of all stakeholders is essential to promote uniformity in future pemphigus outcome measurement and to advance pemphigus drug development.

Background: Biomarkers of low-grade systemic inflammation have been reported to be higher in patients with psoriasis than in healthy controls. However, it is unknown whether this low-grade systemic inflammation contributes to the development of psoriasis or is merely a consequence.

Objectives: To investigate whether low-grade systemic inflammation, measured as systemic immune-inflammation index (SII), neutrophil-to-lymphocyte ratio (NLR) or C-reactive protein (CRP), is an independent risk factor for psoriasis.

Methods: We used data from the Copenhagen General Population Study, a prospective cohort study of the Danish general population where individuals aged 20-100 years were enrolled between 2003 and 2015. Upon enrolment in the study, all individuals underwent a physical examination, completed an extensive self-reported questionnaire regarding lifestyle, and provided blood samples, from which SII, NLR and CRP were measured. Psoriasis was identified using International Classification of Diseases codes with individual linkage to the Danish National Patient Registry. Associations between SII, NLR and CRP and psoriasis were estimated using hazard ratios from Cox proportional hazard regression models. Analyses were adjusted for potential confounders including sex, age, smoking, alcohol consumption, physical activity, educational level, hypertension, dyslipidaemia and obesity.

Results: We included 105 418 individuals with a median age of 58 years, 55% of whom were women. The risk of receiving a diagnosis of psoriasis increased with increasing levels of SII, NLR and CRP. In individuals with high levels (> 90th percentile) of SII, NLR and CRP, the multivariable adjusted hazard ratios were 1.78 [95% confidence interval (CI) 1.41-2.24], 1.56 (95% CI 1.22-1.99) and 2.83 (95% CI 2.27-3.51), respectively, compared with individuals with low levels. Results were similar but slightly attenuated when we used topical calcipotriol (alone or in combination with corticosteroids) for mild psoriasis.

Conclusions: We found that low-grade systemic inflammation, as measured by SII, NLR and CRP, was an independent risk factor for psoriasis, especially moderate-to-severe disease. These findings support the hypothesis that low-grade systemic inflammation may contribute to the pathogenesis of psoriasis rather than simply being a consequence of the disease.

Background: The Comprehensive Quality-of-Life Measure for Acne (CompAQ) is a patient-reported outcome measure (PROM) assessing health-related quality of life (HRQoL) among patients with acne. The measure is one of two acne-specific PROMs that had the strongest evidence to support its use in a recent COSMIN systematic review.

Objectives: To build on prior validation studies of CompAQ by confirming several of its known measurement properties in a cohort of patients with acne at three international sites; and, to establish the responsiveness of the measure during acne treatment.

Methods: Adults being seen for routine acne treatment at dermatology clinics in Canada, the USA and Singapore were recruited between July 2022 and September 2024 to participate in this cohort study. At each visit, participants completed the CompAQ, which consists of 20 items representing five domains (i.e. emotions, social judgement, social interaction, treatment and symptoms domains) and several other relevant measures. Structural validity was assessed by confirmatory factor analysis, internal consistency by calculating Cronbach's α values, and construct validity by known groups and convergent validity hypothesis testing. We further evaluated for differential item functioning (DIF) across study site, sex and age. In addition, we assessed responsiveness by change-score validity hypothesis testing and calculating standardized response means (SRMs).

Results: Our study included 315 participants [mean (SD) age 25.2 (6.1) years; 219 (69.7%) women] and data were collected from a total of 588 visits. The CompAQ was found to have sufficient structural validity, internal consistency and construct validity among patients at each study site and in the combined cohort. In addition, no statistically significant uniform or nonuniform DIF was flagged for most items when analysing DIF by site, sex and age. Based on SRM estimates, the emotions (SRM 0.927) and symptoms (0.914) domains had the largest responsiveness, while the social judgement (0.606), social interaction (0.522) and treatment (0.787) domains had more moderate responsiveness.

Conclusions: CompAQ represents a useful and responsive PROM to evaluate HRQoL among those with acne in clinical trials and clinical practice. Future studies are needed to evaluate the interpretability of the measure (i.e. identifying severity strata and minimally important differences).

The skin is the organ most frequently involved in childhood mastocytosis. In this article, we present a comprehensive update on recent relevant data in the field of childhood mastocytosis, and critically review the drawbacks of its current classification and skin manifestations. We hypothesize that two main types of childhood mastocytosis can be recognized, with relevant implications for the outcome, management and understanding of the disease.