Wanat, Karolyn A.

ImportanceMetastatic cutaneous Crohn disease (MCD) is a rare condition for which there are currently no published diagnostic criteria. Formal MCD diagnostic criteria will expand clinical care and research by enabling future diagnostic code validation, crystallizing a more uniform disease entity for the purposes of translational research, and allowing the development of more formalized outcome measures aimed at treatment response.ObjectiveTo define a set of criteria for the diagnosis of MCD.Evidence ReviewIn this consensus statement, a panel of experts on MCD and related conditions was assembled. Their opinions were gathered regarding the clinical and histologic findings that are sufficient, necessary, and highly suggestive of MCD through serial survey. Subsequent surveys were continued until consensus was achieved. Sufficient was defined as a feature that, if noted, would enable diagnosis of MCD without any other signs, symptoms, or testing. Necessary was defined as a feature that must be present to establish a diagnosis of MCD. Highly suggestive referred to any other features that were indicative of MCD but did not qualify as sufficient or necessary to diagnose MCD.FindingsOf 24 experts invited, 19 agreed to participate, with a 79% to 100% response rate across 5 rounds conducted. Five patients were also invited to participate, but none opted to participate, citing lack of clinical expertise and time concerns. The experts were all from the US; 5 (26%) have practiced medicine for 0 to 5 years, 5 (26%) practiced for 6 to 10 years, 6 (32%) practiced for 11 to 15 years, and 3 (16%) practiced for more than 20 years. A total of 18 experts (95%) worked in an academic practice setting. There was consensus agreement that MCD could present as oral disease, genital disease, or, rarely, in another location. Consensus was achieved on 2 major and 5 minor criteria for genital/other subtypes of MCD and 5 minor criteria for oral MCD. There was consensus that, while there are some pathological findings that are highly suggestive of MCD, histologic criteria are not necessary for diagnosis.Conclusions and RelevanceA panel of experts reached consensus on the diagnostic criteria for MCD described in this study. These criteria include major and minor clinical criteria, along with histopathological criteria for the diagnosis of genital and oral MCD. This consensus is a crucial step in the diagnosis of and future treatment development for MCD, a rare and clinically understudied condition.

Importance Scoring systems for Stevens-Johnson syndrome and epidermal necrolysis (EN) only estimate patient prognosis and are weighted toward comorbidities and systemic features; morphologic terminology for EN lesions is inconsistent. Objectives To establish consensus among expert dermatologists on EN terminology, morphologic progression, and most-affected sites, and to build a framework for developing a skin-directed scoring system for EN. Evidence Review A Delphi consensus using the RAND/UCLA appropriateness criteria was initiated with a core group from the Society of Dermatology Hospitalists to establish agreement on the optimal design for an EN cutaneous scoring instrument, terminology, morphologic traits, and sites of involvement. Findings In round 1, the 54 participating dermatology hospitalists reached consensus on all 49 statements (30 appropriate, 3 inappropriate, 16 uncertain). In round 2, they agreed on another 15 statements (8 appropriate, 7 uncertain). There was consistent agreement on the need for a skin-specific instrument; on the most-often affected skin sites (head and neck, chest, upper back, ocular mucosa, oral mucosa); and that blanching erythema, dusky erythema, targetoid erythema, vesicles/bullae, desquamation, and erosions comprise the morphologic traits of EN and can be consistently differentiated. Conclusions and Relevance This consensus exercise confirmed the need for an EN skin-directed scoring system, nomenclature, and differentiation of specific morphologic traits, and identified the sites most affected. It also established a baseline consensus for a standardized EN instrument with consistent terminology.

ImportanceMorphea is an insidious inflammatory disorder of the skin and deeper tissues. Determining disease activity is challenging yet important to medical decision-making and patient outcomes.ObjectiveTo develop and validate a scoring tool, the Morphea Activity Measure (MAM), to evaluate morphea disease activity of any type or severity that is easy to use in clinical and research settings.Design, Setting, and ParticipantsThis pilot diagnostic study was conducted from September 9, 2019, to March 6, 2020, in 2 phases: development and validation. During the development phase, 14 morphea experts (dermatologists and pediatric dermatologists) used a Delphi consensus method to determine items that would be included in the MAM. The validation phase included 8 investigators who evaluated the tool in collaboration with 14 patients with pediatric morphea (recruited from a referral center [Medical College of Wisconsin]) during a 1-day in-person meeting on March 6, 2020.Main Outcomes and MeasuresDuring the development phase, online survey items were evaluated by experts in morphea using a Likert scale (score range, 0-10, with 0 indicating not important and 10 indicating very important); agreement was defined as a median score of 7.0 or higher, disagreement as a median score of 3.9 or lower, and no consensus as a median score of 4.0 to 6.9. During the validation phase, reliability (interrater and intrarater agreement using intraclass correlation coefficients), validity (using the content validity index and κ statistics as well as correlations with the modified Localized Scleroderma Severity Index and the Physician Global Assessment of Activity using Spearman ρ coefficients), and viability (using qualitative interviews of investigators who used the MAM tool) were evaluated. Descriptive statistics were used for quantitative variables. Data on race and ethnicity categories were collected but not analyzed because skin color was more relevant for the purposes of this study.ResultsAmong 14 survey respondents during the development phase, 9 (64.3%) were pediatric dermatologists and 5 (35.7%) were dermatologists. After 2 rounds, a final tool was developed comprising 10 items that experts agreed were indicative of morphea activity (new lesion in the past 3 months, enlarging lesion in the past 3 months, linear lesion developing progressive atrophy in the past 3 months, erythema, violaceous rim or color, warmth to the touch, induration, white-yellow or waxy appearance, shiny white wrinkling, and body surface area). The validation phase was conducted with 14 patients (median age, 14.5 years [range, 8.0-18.0 years]; 8 [57.1%] female), 2 dermatologists, and 6 pediatric dermatologists. Interrater and intrarater agreement for MAM total scores was good, with intraclass correlation coefficients of 0.844 (95% CI, 0.681-0.942) for interrater agreement and 0.856 (95% CI, 0.791-0.901) for intrarater agreement. Correlations between the MAM and the modified Localized Scleroderma Severity Index (Spearman ρ = 0.747; P < .001) and the MAM and the Physician Global Assessment of Activity (Spearman ρ = 0.729; P < .001) were moderately strong. In qualitative interviews, evaluators agreed that the tool was easy to use, measured morphea disease activity at a single time point, and should be responsive to changes in morphea disease activity over multiple time points.Conclusions and RelevanceIn this study, the MAM was found to be a reliable, valid, and viable tool to measure pediatric morphea activity. Further testing to assess validity in adults and responsiveness to change is needed.