Faculty Opinions recommendation of Development and initial validation of the localized scleroderma skin damage index and physician global assessment of disease damage: a proof-of-concept study.

Objective.Pansclerotic morphea is a poorly described form of morphea with little information on prevalence, demographics, and clinical features. Classification criteria for this subtype varies and the distinction from other forms of morphea, such as extensive generalized morphea and pansclerotic morphea, is not always clear. The purpose of our study was to clarify classification criteria for pansclerotic morphea by identifying its prevalence in the morphea in adults and children (MAC) cohort and describing its demographic and clinical features as compared with generalized morphea.Methods.Patients who met predefined criteria for generalized and pansclerotic morphea were identified using a modified Laxer and Zulian classification system. Baseline demographic and clinical features of the patients were compiled and then analyzed for traits characteristic of pansclerotic morphea versus those of generalized morphea. One hundred and thirteen patients met the criteria for inclusion: pansclerotic (n = 13) and generalized morphea type (n = 100).Results.Patients with pansclerotic morphea were more frequently male (46.2% vs 6%; p < 0.0001); had a shorter time to diagnosis (mean difference of 10.4 mos; 95% CI: 0.8–19.9 mos; p = 0.0332); higher rates of functional impairment (61.5% vs 16%; p = 0.0046); higher rates of deep involvement (61.5% vs 17%; p = 0.004); and higher average Rodnan Skin Score (mean difference of 10.8 points; 95% CI: 5–16.6; p = 0.0017), Localized Scleroderma Skin Damage Index (mean difference 28.3; 95% CI: 9–47.6; p = 0.009), and Physician Global Assessment of Disease Damage scores (mean difference 25.1; 95% CI: 0.3–50; p = 0.048).Conclusion.Our results suggest demographic and clinical features are sufficient to define the pansclerotic subtype as they represent a distinct clinical phenotype with a more rapidly progressive and severe course commonly accompanied by disability. Presence of features of the pansclerotic phenotype should alert practitioners to the possibility of significant morbidity and the need for early aggressive treatment.

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In silico validation of the Autoinflammatory Disease Damage Index

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2018-213725 ◽

2018 ◽

Vol 77 (11) ◽

pp. 1599-1605 ◽

Cited By ~ 10

Author(s):

Nienke M ter Haar ◽

Amber Laetitia Justine van Delft ◽

Kim Valerie Annink ◽

Henk van Stel ◽

Sulaiman M Al-Mayouf ◽

...

Keyword(s):

Disease Activity ◽

Autoinflammatory Disease ◽

Intraclass Correlation ◽

Damage Index ◽

Autoinflammatory Diseases ◽

Tumour Necrosis Factor Receptor ◽

Mevalonate Kinase Deficiency ◽

Physician Global Assessment ◽

Disease Damage ◽

Clinical Cases

IntroductionAutoinflammatory diseases can cause irreversible tissue damage due to systemic inflammation. Recently, the Autoinflammatory Disease Damage Index (ADDI) was developed. The ADDI is the first instrument to quantify damage in familial Mediterranean fever, cryopyrin-associated periodic syndromes, mevalonate kinase deficiency and tumour necrosis factor receptor-associated periodic syndrome. The aim of this study was to validate this tool for its intended use in a clinical/research setting.MethodsThe ADDI was scored on paper clinical cases by at least three physicians per case, independently of each other. Face and content validity were assessed by requesting comments on the ADDI. Reliability was tested by calculating the intraclass correlation coefficient (ICC) using an ‘observer-nested-within-subject’ design. Construct validity was determined by correlating the ADDI score to the Physician Global Assessment (PGA) of damage and disease activity. Redundancy of individual items was determined with Cronbach’s alpha.ResultsThe ADDI was validated on a total of 110 paper clinical cases by 37 experts in autoinflammatory diseases. This yielded an ICC of 0.84 (95% CI 0.78 to 0.89). The ADDI score correlated strongly with PGA-damage (r=0.92, 95% CI 0.88 to 0.95) and was not strongly influenced by disease activity (r=0.395, 95% CI 0.21 to 0.55). After comments from disease experts, some item definitions were refined. The interitem correlation in all different categories was lower than 0.7, indicating that there was no redundancy between individual damage items.ConclusionThe ADDI is a reliable and valid instrument to quantify damage in individual patients and can be used to compare disease outcomes in clinical studies.

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The Localized Scleroderma Skin Severity Index and Physician Global Assessment of Disease Activity: A Work in Progress Toward Development of Localized Scleroderma Outcome Measures

Yearbook of Dermatology and Dermatologic Surgery ◽

10.1016/s0093-3619(10)79709-4 ◽

2011 ◽

Vol 2011 ◽

pp. 224-225

Author(s):

B.P. Glick

Keyword(s):

Disease Activity ◽

Outcome Measures ◽

Global Assessment ◽

Severity Index ◽

Localized Scleroderma ◽

Physician Global Assessment ◽

Work In Progress ◽

Assessment Of Disease Activity

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Extracutaneous Involvement Is Common and Associated with Prolonged Disease Activity and Greater Impact in Juvenile Localized Scleroderma

Rheumatology ◽

10.1093/rheumatology/keab238 ◽

2021 ◽

Author(s):

Suzanne C Li ◽

Gloria C Higgins ◽

Mallory Chen ◽

Kathryn S Torok ◽

C Egla Rabinovich ◽

...

Keyword(s):

Disease Activity ◽

Global Assessment ◽

Disease Onset ◽

Response To Treatment ◽

Localized Scleroderma ◽

Physician Global Assessment ◽

Treatment Needs ◽

Organ Systems ◽

Disease Impact ◽

Juvenile Localized Scleroderma

Abstract Objective To evaluate factors associated with extracutaneous involvement (ECI) in juvenile localized scleroderma (jLS). Methods A prospective, multi-center, 6-month observational study was performed. Collected data included disease features, global assessments, and subject symptoms. Bivariate and linear multilevel regression analyses were performed. Results Eighty-six jLS subjects (80% female, 80% Caucasian), median age of disease onset 7.7 years, were evaluated. Most had linear scleroderma or mixed morphea. Fourty-nine subjects (57%) had 125 extracutaneous problems (median 2 (IQR 1, 3) per subject) from 9 organ systems. Most of these subjects had multiple musculoskeletal problems. ECI was associated with more extensive cutaneous involvement, higher number of symptoms, family history of autoimmunity, and ANA and rheumatoid factor positivity. Subjects with ECI had higher scores for physician global assessment of damage (PGA-D), and parental global assessment of disease impact, but not baseline physician global assessment of disease activity (PGA-A). Although subjects with ECI received more methotrexate and glucocorticoid treatment, they had a slower reduction in PGA-A scores and symptoms over time, suggesting a poorer response to treatment. In logistic regression modeling, female sex had the largest effect on parental impact scores. Conclusions ECI occurred in the majority of subjects with jLS, and was associated with more medication use, longer treatment duration, higher PGA-D scores, and higher parental assessment of disease impact. Our findings suggest that jLS subjects with ECI have greater overall disease burden, both cutaneous and extracutaneous, and poorer response to treatment. More study of the treatment needs of this population is warranted.

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THU0280 IMPACT ON PHYSICIAN GLOBAL ASSESSMENT ON REMISSION RATES IN SLE. ANALYSIS FROM A GERMAN SLE-COHORT.

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2020-eular.3860 ◽

2020 ◽

Vol 79 (Suppl 1) ◽

pp. 367.2-367

Author(s):

J. Mucke ◽

C. Duesing ◽

M. Schneider ◽

G. Chehab

Keyword(s):

Immunosuppressive Therapy ◽

Global Assessment ◽

Rating Scale ◽

Task Force ◽

Damage Index ◽

Depression Scale ◽

Added Value ◽

Physician Global Assessment ◽

Research Support ◽

Number Of Patients

Background:Defining remission for SLE as a suitable target for a treat to target (T2T) approach has been a major challenge in the past years. A few years back, four definitions of remission were presented by the international DORIS task force.[1] Parameters included in the definition are clinical activity (cSLEDAI), steroid dose, immunosuppressive therapy, serology and physician global assessment (PGA). In particular the PGA, its threshold and general utility have been and still are discussed controversially.Objectives:It was our aim to evaluate the added value of PGA in remission assessment.Methods:In this monocentric cross-sectional study, patients with SLE according to the 1997 American College of Rheumatology (ACR) criteria were enrolled and assessed between September 2016 and December 2017. Two different definitions of remission were applied. The internationally accepted DORIS remission and a modified DORIS remission excluding PGA. Factors influencing PGA were assessed in the entire cohort. Regression analyses were used to assess differences between patients in DORIS and modified DORIS remission.Results:A total of 233 patients were included (87.6% female). 98 patients (41.9%) fulfilled any of the four DORIS remission definitions, while 154 patients (66.1%) were in any modified remission in which PGA was excluded. In general, PGA rating was associated with disease activity (clinical SLEDAI; p=<0.0001), depression (Center for Epidemiological Studies Depression Scale; p=0.049), pain reported by the patient (numeric rating scale; p=<0.0001) and hypocomplementemia (p=<0.0001). Damage (SLICC damage index, SDI) did not influence PGA (p=0.98). Both, DORIS and modified DORIS remission were associated with lower damage (p=0.026; p=0.003), lower pain on NRS (p=0.001; p=0.013), normal complement (p=0.0005; p=0.005) and better illness perception (p=0.006; p=0.023). Patients in modified DORIS remission had a tendency for more immunosuppressive therapy (p=0.046).Conclusion:Exclusion of PGA in remission assessment led to an increased number of patients in remission. Clinical parameters and factors associated with DORIS remission vs. modified DORIS remission were similar, hence the added value of PGA in our cohort regarding remission assessment is questionable. The use and especially the correct threshold of PGA for remission still has to be discussed.References:[1]van Vollenhoven, Ronald; Voskuyl, Alexandre; Bertsias, George; Aranow, Cynthia; Aringer, Martin; Arnaud, Laurent et al. (2017): A framework for remission in SLE: consensus findings from a large international task force on definitions of remission in SLE (DORIS). In:Annals of the rheumatic diseases76 (3), S. 554–561. DOI: 10.1136/annrheumdis-2016-209519.Disclosure of Interests:Johanna Mucke: None declared, Christina Duesing: None declared, Matthias Schneider Grant/research support from: GSK, UCB, Abbvie, Consultant of: Abbvie, Alexion, Astra Zeneca, BMS, Boehringer Ingelheim, Gilead, Lilly, Sanofi, UCB, Speakers bureau: Abbvie, Astra Zeneca, BMS, Chugai, GSK, Lilly, Pfizer, Sanofi, Gamal Chehab Grant/research support from: Grant/research support from: GlaxoSmithKline and UCB Pharma for performing the LuLa-study.

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The Localized Scleroderma Skin Severity Index and Physician Global Assessment of Disease Activity: A Work in Progress Toward Development of Localized Scleroderma Outcome Measures

The Journal of Rheumatology ◽

10.3899/jrheum.081284 ◽

2009 ◽

Vol 36 (12) ◽

pp. 2819-2829 ◽

Cited By ~ 97

Author(s):

THASCHAWEE ARKACHAISRI ◽

SOAMARAT VILAIYUK ◽

SUZANNE LI ◽

KATHLEEN M. O’NEIL ◽

ELENA POPE ◽

...

Keyword(s):

Disease Activity ◽

Disease Severity ◽

Outcome Measures ◽

Global Assessment ◽

Severity Index ◽

Localized Scleroderma ◽

Second Phase ◽

Physician Global Assessment ◽

Intrarater Reliability ◽

Assessment Of Disease Activity

Objective.To develop and evaluate a Localized Scleroderma (LS) Skin Severity Index (LoSSI) and global assessments’ clinimetric property and effect on quality of life (QOL).Methods.A 3-phase study was conducted. The first phase involved 15 patients with LS and 14 examiners who assessed LoSSI [surface area (SA), erythema (ER), skin thickness (ST), and new lesion/extension (N/E)] twice for inter/intrarater reliability. Patient global assessment of disease severity (PtGA-S) and Children’s Dermatology Life Quality Index (CDLQI) were collected for intrarater reliability evaluation. The second phase was aimed to develop clinical determinants for physician global assessment of disease activity (PhysGA-A) and to assess its content validity. The third phase involved 2 examiners assessing LoSSI and PhysGA-A on 27 patients. Effect of training on improving reliability/validity and sensitivity to change of the LoSSI and PhysGA-A was determined.Results.Interrater reliability was excellent for ER [intraclass correlation coefficient (ICC) 0.71], ST (ICC 0.70), LoSSI (ICC 0.80), and PhysGA-A (ICC 0.90) but poor for SA (ICC 0.35); thus, LoSSI was modified to mLoSSI. Examiners’ experience did not affect the scores, but training/practice improved reliability. Intrarater reliability was excellent for ER, ST, and LoSSI (Spearman’s rho = 0.71–0.89) and moderate for SA. PtGA-S and CDLQI showed good intrarater agreement (ICC 0.63 and 0.80). mLoSSI correlated moderately with PhysGA-A and PtGA-S. Both mLoSSI and PhysGA-A were sensitive to change following therapy.Conclusion.mLoSSI and PhysGA-A are reliable and valid tools for assessing LS disease severity and show high sensitivity to detect change over time. These tools are feasible for use in routine clinical practice. They should be considered for inclusion in a core set of LS outcome measures for clinical trials.

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