scholarly journals Preliminary Validation of the Digital Ulcer Clinical Assessment Score in Systemic Sclerosis

2018 ◽  
Vol 46 (6) ◽  
pp. 603-608 ◽  
Author(s):  
Cosimo Bruni ◽  
Tanaka Ngcozana ◽  
Francesca Braschi ◽  
Tiziana Pucci ◽  
Guya Piemonte ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Construct Validity ◽  
Clinical Assessment ◽  
Hand Function ◽  
Assessment Tools ◽  
Face Validity ◽  
Pain Patient ◽  
Digital Ulcers ◽  
Assessment Score ◽  
Patient Reported

Objective.To date, “healed/non-healed” and clinical judgment are the only available assessment tools for digital ulcers (DU) in patients with systemic sclerosis (SSc). The aim of our study is to examine a preliminary composite DU clinical assessment score (DUCAS) for SSc for face, content, and construct validity.Methods.Patients with SSc presenting at least 1 finger DU were enrolled and assessed with the Health Assessment Questionnaire–Disability Index, Cochin scale, visual analog scale (VAS) for DU-related pain, patient global DU status, and global assessment as patient-reported outcomes (PRO), and physician VAS for DU status (phyGDU) as an SSc-DU expert physician/nurse measure. The DUCAS included 7 DU-related variables selected by a committee of SSc DU experts and weighted on a clinical basis. Face validity was examined by consensus and partial construct validity was tested through convergent correlation with other measures of hand function, using Spearman’s correlations. A range of patients with SSc was examined. A linear regression model with backward stepwise analysis was used to determine the relationship of individual variables with the primary clinical parameter, phyGDU.Results.Forty-four patients with SSc (9 males, mean age 55 ± 15 yrs, mean disease duration 9.9 ± 5.8 yrs) were enrolled in the study. Overall DUCAS showed significant positive correlations with all abovementioned PRO (r > 0.4, p < 0.01). When all scores and scales were modeled, only DUCAS significantly predicted phyGDU (r = 0.59, R2 = 0.354, Akaike information criterion = 385.4).Conclusion.Preliminarily, we suggest that the DUCAS may be a new clinical score for SSc-related DU, having face and content validity and convergent/divergent correlations (construct validity). These early data suggest that this score deserves further evaluation.

scholarly journals Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument

2020 ◽  
Vol 22 (1) ◽  
Author(s):  
Luc Mouthon ◽  
Serge Poiraudeau ◽  
Margaret Vernon ◽  
Kelly Papadakis ◽  
Loïc Perchenet ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Construct Validity ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
Digital Ulcers ◽  
Meaningful Change ◽  
Patient Reported ◽  
Psychometric Analyses ◽  
Hand Disability ◽  
Mean Differences

Abstract Background We aimed to develop a patient-reported outcome measure, in accordance with the US Food and Drug Administration guidance, to capture the impact of systemic sclerosis-related digital ulcers (SSc-DUs) on hand function. Psychometric analyses were conducted to evaluate and document the measurement properties of the resulting instrument—the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®). Methods The HDISS-DU was developed through a series of confirmatory, qualitative concept-elicitation interviews (N = 36) to provide supportive evidence that the instrument captures all relevant issues and functional limitations relating to SSc-DUs in this patient population. Psychometric analyses used blinded data from two randomised, controlled, phase 3 trials in patients with SSc-DUs (N = 517). The analyses included assessment of reliability, construct validity, responsiveness and thresholds for meaningful change. Results Qualitative interviews confirmed that the HDISS-DU had good content coverage and patients understood the HDISS-DU instructions, items and response scale. The HDISS-DU demonstrated excellent internal consistency and test-retest reliability, with satisfactory construct validity. Overall, the HDISS-DU was highly responsive to change in digital ulcer severity: the no-change group (for other criterion measures) had mean differences and effect sizes close to 0, while mean differences were mostly negative (indicating improvement) for the improvement groups (for other criterion measures) and vice versa. The preliminary threshold for meaningful change was a 0.50 difference in HDISS-DU score. Conclusions Using data from two large studies of SSc-DU patients, these psychometric analyses support the reliability, validity, discriminating ability and responsiveness to change of the HDISS-DU for evaluating treatment outcomes in future clinical studies and clinical practice.

scholarly journals Digital Ulcers as Presentation of Carpal Tunnel Syndrome

2020 ◽  
Vol 77 (4) ◽  
pp. 353-356
Author(s):  
T. F. Gomes ◽  
K Kieselova ◽  
F. Santiago ◽  
A. Daniel ◽  
M. Henrique
Keyword(s):  
Systemic Sclerosis ◽  
Carpal Tunnel Syndrome ◽  
Carpal Tunnel ◽  
Clinical Assessment ◽  
Gold Standard ◽  
Digital Ulcers ◽  
Tunnel Syndrome ◽  
Sensory Changes ◽  
Hand Radiography

Ulcerating and mutilating variant of carpal tunnel syndrome occurs in longstanding cases of untreated disease. Pa- tients present with painless ulcers of the second and third fingers, accompanied with other cutaneous and sensory changes. These patients are often misdiagnosed as having a Raynaud disease or systemic sclerosis. Clinical assessment is the gold standard for the diagnosis of carpal tunnel syndrome, but hand radiography and electromyography help supporting the diagnosis. The authors present two cases of this ulcerating variant of carpal tunnel syndrome.

Digital ulcers in systemic sclerosis: role of flow-mediated dilatation and capillaroscopy as risk assessment tools

2015 ◽  
Vol 25 (5) ◽  
pp. 444-451 ◽  
Author(s):  
Ivone Silva ◽  
Tiago Loureiro ◽  
Andreia Teixeira ◽  
Isabel Almeida ◽  
Armando Mansilha ◽  
...  
Keyword(s):  
Risk Assessment ◽  
Systemic Sclerosis ◽  
Assessment Tools ◽  
Digital Ulcers ◽  
Risk Assessment Tools ◽  
Flow Mediated Dilatation

Validity of the PROMIS-29 in a large Australian cohort of patients with systemic sclerosis

2017 ◽  
Vol 2 (3) ◽  
pp. 188-195 ◽  
Author(s):  
Kathleen Morrisroe ◽  
Wendy Stevens ◽  
Molla Huq ◽  
Joanne Sahhar ◽  
Gene-Siew Ngian ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Systemic Sclerosis ◽  
Construct Validity ◽  
Short Form ◽  
Good Alternative ◽  
Measurement Information ◽  
Internal Reliability ◽  
Sf 36 ◽  
Patient Reported

Background We aimed to evaluate the construct validity of the Patient-Reported Outcomes Measurement Information System 29 (PROMIS-29) in Australian systemic sclerosis (SSc) patients. Methods SSc patients, identified through the Australian Scleroderma Cohort Study database, completed two quality-of-life instruments concurrently, the PROMIS-29 and the Medical Outcomes Study 36-Item Short Form Health Survey (SF-36). The construct validity of the PROMIS-29 was assessed by the correlations between the PROMIS-29 and the SF-36 and Health Assessment Questionnaire Disability Index (HAQ-DI). Cronbach's alpha was used to test the internal reliability of all instruments in Australian SSc patients and non-parametric correlation, including Spearman's correlation, was used to test the construct validity of PROMIS-29 against the SF-36 and HAQ-DI. Results A total of 477 completed questionnaires were returned, equating to a response rate of 59.6%. The mean (±SD) age of respondents at the time of the survey was 64.1 (±11.1) years. They were predominantly female (87.4%), with limited disease subtype (lcSSc) (77.8%) and long disease duration from onset of first non-Raynaud's phenomenon symptom at the time of survey (10.9 ± 11.1 years). For the correlation analysis between the PROMIS-29 and the legacy instruments, all Spearman correlation coefficients were in the logical direction and highly significant suggesting that the PROMIS-29 is a good alternative to other validated measures of disease burden. Conclusions Our study indicates that the PROMIS-29 questionnaire is a valid instrument for measuring health-related quality of life in Australian females with lcSSc of long duration.

scholarly journals ADIPOSE-DERIVED STROMAL VASCULAR FRACTION AND FAT GRAFTS VERSUS MEDICAL TREATMENT FOR TREATING THE HANDS OF PATIENTS WITH SYSTEMIC SCLEROSIS. A RANDOMIZED CRONTOLLED TRIAL

2021 ◽  
Author(s):  
Martin Iglesias ◽  
Iván Torre-Villalvazo ◽  
Patricia Butrón-Gandarillas ◽  
Tatiana S. Rodríguez-Reyna ◽  
Erik A. Torre-Anaya ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Systemic Sclerosis ◽  
Medical Treatment ◽  
Disease Severity ◽  
Hand Function ◽  
Stromal Vascular Fraction ◽  
Control Group ◽  
Digital Ulcers ◽  
Experimental Group

ABSTRACTBackgroundIn the hand, the Systemic Sclerosis (SS) is characteristically evidenced by Raynaud’s phenomenon (RP) and fibrosis of the skin, tendons, ligaments, and joints as well as digital ulcers with prolonged healing. Current medical treatment not always cure these complications. Local adipose-derived stromal vascular fraction (ADSVF) administration into the hands has been proposed as an emerging treatment for these complications, due to its proangiogenic, antifibrotic, and immunoregulatory activities. The objective of this controlled trial was to evaluate the safety and clinical effects of fat micrografts plus ADSVF administration into the hands of patients with SS.MethodsThis was an open-label, monocentric, randomized controlled study. Twenty patients diagnosed with SS were enrolled and assigned to the experimental or control group. Fat micrografts plus the ADSVF were injected into the right hand of experimental group patients. The control group continued to receive only medical treatment. Demographic, serologic data and disease severity were recorded. Digital oximetry, pain, Raynaud phenomenon (RP), digital ulcer healing (DUH), mobility, thumb opposition, vascular density of the nail bed, skin affection of the hand, Serologic antibodies, hand function, and quality of life scores were evaluated in both groups. The mean follow-up period was 168 days.The differences between before and after the intervention were analyzed with the Wilcoxon range test, and the differences between the control and experimental groups at 0 days and 168 days were analyzed with the Mann–Whitney U test.ResultsAdverse events were not observed in both groups. There were no changes in disease severity, serologic antibodies, nailfold capillaroscopy patterns, mobility, and hand function in both groups. There were significant improvements in pain, DUH and quality of life scores in the experimental group. RP improved significantly in both groups. However, on statistically comparing the results at 168 days between the groups, significant improvements were only observed in pain levels (p = 0.02) and DUH (p=0.003).ConclusionsThe injection of ADSVF plus fat micrografts is a reproducible, and safe technique. Pain and digital ulcers in the hands of patients with SS can be treated with this treatment.Trial RegistrationRetrospectively registered in ClinicalTrials.gov with identifier NCT04387825

AB0786 Hand function and disability in patients with systemic sclerosis and digital ulcers:

2013 ◽  
Vol 71 (Suppl 3) ◽  
pp. 683.10-683
Author(s):  
A. Nitsche ◽  
C. Amitrano ◽  
P. Pucci ◽  
A. Lescano
Keyword(s):  
Systemic Sclerosis ◽  
Hand Function ◽  
Digital Ulcers

scholarly journals SAT0321 CURRENT PATIENT REPORTED OUTCOMES (PROS) POORLY REFLECT CHANGES IN LUNG FUNCTION IN PATIENTS WITH SYSTEMIC SCLEROSIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1106.1-1106
Author(s):  
F. Danzo ◽  
K. Gjeloshi ◽  
G. Abignano ◽  
A. M. Dean ◽  
F. Masini ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Lung Function ◽  
Health Assessment Questionnaire ◽  
Patient Reported Outcomes ◽  
Health Assessment ◽  
Digital Ulcers ◽  
Strong Positive Correlation ◽  
Patient Reported ◽  
Assessment Questionnaire

Background:Lung involvement is very common is systemic sclerosis (SSc). Approximately one quarter of patients develops pulmonary problems within the first 3 years of diagnosis and still represents the leading cause of death in these patients. In a recent clinical trail, the reduction of FVC was not accompanied by a benefit with respect to health-related quality of life and patient-reported outcomes (PROs).Objectives:To assess how the change in Pulmonary Function Test (PFTs) parameters correlates with the Patient Reported Outcomes (PROs) in an observational cohort of patients with Systemic Sclerosis (SSc).Methods:We conducted a retrospective study of 330 clinic episodes from 121 unselected patients diagnosed with systemic sclerosis according to EULAR/ACR 2013 criteria, in annual follow-up (for a total of 165 patients/year) with PFTs, Health Assessment Questionnaire Disability Index (HAQ-DI), Scleroderma Health Assessment Questionnaire (sHAQ), Modified Borg Dyspnea Scale (Borg) and Cochin Hand Function Score (CHFS). We assessed the correlation between the HAQ and the Visual Analogical Scale 1-7 at baseline (VAS1 pain, VAS2 disease severity, VAS3 arthritis activity, VAS4 intestinal problems, VAS5 dyspnea, VAS6 Raynaud’s phenomenon, VAS7 digital ulcers). We evaluated the correlation of PFTs with PROs at every time period and the correlation between the change of PFTs parameters (δFVC, δDLCO) with the change of the PROs over a year of follow-up. Following analysis of distribution, Spearman or Pearson Test were used to determine correlation coefficients, as appropiate (Prism 7).Results:The median disease duration was 5 years (IQR 3-10). The median of 12 months δFVC% and δDLCO% were 0 (IQR -5.81 to 3.28) and -2.439 (IQR -8.76 to 5.98), respectively. The analysis evidenced a strong positive correlation between VAS1-7 and HAQ. We observed also significant correlation between FVC%, DLCO% and HAQ-DI (r= - 0.355 and -0.266, respectively; p<0.0001 for both), Borg (r= -0.403 and -0.379, respectively; p<0.0001) and CHFS (r = -0.355 and -0.256, respectively; p<0.0001). Nevertheless, in longitudinal setting there was no significant correlation between δPROs and changes lung function, as continuous variables, neither there was any significant PROs difference in patients that did or did not lose more than 10% of FVC and DLCO over a year of follow-up.Conclusion:This analysis of a monocentric non-selected population evidenced that the current commonly used PROs in SSc while showing a good correlation with lung function are poorly sensitive to change or to reflect changes in lung function over 12 months. In this sense, prudent interpretation of the lack of correlation between FVC and patient-reported outcomes in studies of phase 3 is warranted.References:[1]Rahimi S., Nintedanib for systemic sclerosis-associated interstitial lung disease, Lancet Respiratory Medicine (2020)Disclosure of Interests:None declared

scholarly journals OP0251 THE EULAR SYSTEMIC SCLEROSIS IMPACT OF DISEASE (SCLEROID) SCORE – A NEW PATIENT-REPORTED OUTCOME MEASURE FOR PATIENTS WITH SYSTEMIC SCLEROSIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 158-159
Author(s):  
M. O. Becker ◽  
R. Dobrota ◽  
K. Fligelstone ◽  
A. Roennow ◽  
Y. Allanore ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Systemic Sclerosis ◽  
Validation Study ◽  
Hand Function ◽  
Patient Reported Outcome ◽  
Sensitivity To Change ◽  
Research Support ◽  
Patient Reported ◽  
Disease Impact

Background:Patient reported outcome measures (PROM) are important for clinical practice and research. Given the unmet need for a comprehensive PROM for systemic sclerosis (SSc), the ScleroID questionnaire was developed by a joint team of patients with SSc and medical experts. This is intended as a brief, specific, patient-derived, disease impact score for research and clinical use in SSc.Objectives:Here, we present the validation and final version of the ScleroID.Methods:This EULAR-endorsed project involves 9 European expert SSc centers. Patients fulfilling the ACR/EULAR 2013 criteria were prospectively included since 05/16 in a large observational cohort study. Patients completed the ScleroID and comparators SHAQ, EQ5D, SF36. They also weighted the 10 dimensions of the ScleroID by distributing 100 points according to the perceived impact on their health. The final score calculation is based on the ranking of the weights. The validation study included a reliability arm and a longitudinal arm, looking at sensitivity to change at follow-up.Results:Of the 472 patients included at baseline, 109 patients also had a reliability visit and 113 patients a follow-up visit. 84.5% of patients were female, 29.8% had diffuse SSc, mean age was 54.6 years, and mean disease duration 9.5 years. The highest weights were assigned by the patients to Raynaud`s phenomenon, fatigue, hand function and pain, confirming our previous results. The total ScleroID score showed good Spearman correlation coefficients with the comparators (SHAQ, 0.73; EQ5D -0.48; Patient’s global assessment, VAS 0.77; HAQ-DI 0.62; SF36 physical score -0.62; each p<0.001). The internal consistency was good: Crohnbach’s alpha 0.866, similar to SS-HAQ (0.88) and higher than EQ5D (0.77). The ScleroID had a very good reliability: intra-class correlation coefficient 0.839 (ranging 0.608 to 0.788 for the individual items), superior to all comparators. Twenty of 113 patients reported a change in their disease status at follow up. Sensitivity to change: the standardized response mean was 0.34 for the total ScleroID score and highest for lower GI (0.633) and life choices domains (0.521), superior to all other PROM. Figure 1 shows the final ScleroID.Figure 1.Conclusion:The EULAR ScleroID is a novel PROM designed for use in clinical practice and clinical trials to reflect the disease impact of SSc, showing good performance in the validation study. Importantly, Raynaud syndrome, impaired hand function, pain and fatigue were the main patient reported drivers of disease impact.Disclosure of Interests:Mike O. Becker: None declared, Rucsandra Dobrota: None declared, Kim Fligelstone: None declared, Annelise Roennow: None declared, Yannick Allanore Grant/research support from: BMS, Inventiva, Roche, Sanofi, Consultant of: Actelion, Bayer AG, BMS, BI, Patricia Carreira Grant/research support from: Actelion, Roche, MSD, Consultant of: GlaxoSmithKline, VivaCell Biotechnology, Emerald Health Pharmaceuticals, Boehringer Ingelheim, Roche, Speakers bureau: Actelion, GlaxoSmithKline, Roche, László Czirják Consultant of: Actelion, BI, Roche-Genentech, Lilly, Medac, Novartis, Pfizer, Bayer AG, Christopher Denton Grant/research support from: GlaxoSmithKline, CSL Behring, and Inventiva, Consultant of: Medscape, Roche-Genentech, Actelion, GlaxoSmithKline, Sanofi Aventis, Inventiva, CSL Behring, Boehringer Ingelheim, Corbus Pharmaceuticals, Acceleron, Curzion and Bayer, Roger Hesselstrand: None declared, Gunnel Sandqvist: None declared, Otylia Kowal-Bielecka Consultant of: Bayer, Boehringer Ingelheim, Inventiva, MSD, Medac, Novartis, Roche and Sandoz, Speakers bureau: Bayer, Boehringer Ingelheim, Inventiva, MSD, Medac, Novartis, Roche and Sandoz, Cosimo Bruni Speakers bureau: Actelion, Eli Lilly, Marco Matucci Cerinic: None declared, Carina Mihai: None declared, Ana Maria Gheorghiu: None declared, Ulf Müller-Ladner Speakers bureau: Biogen, Joe Sexton: None declared, Turid Heiberg: None declared, Oliver Distler Grant/research support from: Grants/Research support from Actelion, Bayer, Boehringer Ingelheim, Competitive Drug Development International Ltd. and Mitsubishi Tanabe; he also holds the issued Patent on mir-29 for the treatment of systemic sclerosis (US8247389, EP2331143)., Consultant of: Consultancy fees from Actelion, Acceleron Pharma, AnaMar, Bayer, Baecon Discovery, Blade Therapeutics, Boehringer, CSL Behring, Catenion, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Sanofi and UCB, Speakers bureau: Speaker fees from Actelion, Bayer, Boehringer Ingelheim, Medscape, Pfizer and Roche

scholarly journals Adipose-Derived Stromal Vascular Fraction and Fat Grafts Versus Medical Treatment for Treating the Hands of Patients With Systemic Sclerosis. A Randomized Crontolled Trial

2021 ◽  
Author(s):  
Martin Iglesias ◽  
Ivan Torre-Villalvazo ◽  
Patricia Butrón-Gandarillas ◽  
Tatiana S Rodríguez-Reyna ◽  
Erik A Torre-Anaya ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Systemic Sclerosis ◽  
Medical Treatment ◽  
Disease Severity ◽  
Hand Function ◽  
Stromal Vascular Fraction ◽  
Control Group ◽  
Digital Ulcers ◽  
Experimental Group

Abstract Background. In the hand, the Systemic Sclerosis (SS) is characteristically evidenced by Raynaud's phenomenon (RP) and fibrosis of the skin, tendons, ligaments, and joints as well as digital ulcers with prolonged healing. Current medical treatment not always cure these complications. Local adipose-derived stromal vascular fraction (ADSVF) administration into the hands has been proposed as an emerging treatment for these complications, due to its proangiogenic, antifibrotic, and immunoregulatory activities. The objective of this controlled trial was to evaluate the safety and clinical effects of fat micrografts plus ADSVF administration into the hands of patients with SS.Methods. This was an open-label, monocentric, randomized controlled study. Twenty patients diagnosed with SS were enrolled and assigned to the experimental or control group. Fat micrografts plus the ADSVF were injected into the right hand of experimental group patients. The control group continued to receive only medical treatment. Demographic, serologic data and disease severity were recorded. Digital oximetry, pain, Raynaud phenomenon (RP), digital ulcer healing (DUH), mobility, thumb opposition, vascular density of the nail bed, skin affection of the hand, Serologic antibodies, hand function, and quality of life scores were evaluated in both groups. The mean follow-up period was 168 days. The differences between before and after the intervention were analyzed with the Wilcoxon range test, and the differences between the control and experimental groups at 0 days and 168 days were analyzed with the Mann–Whitney U test.Results. Adverse events were not observed in both groups. There were no changes in disease severity, serologic antibodies, nailfold capillaroscopy patterns, mobility, and hand function in both groups. There were significant improvements in pain, DUH and quality of life scores in the experimental group. RP improved significantly in both groups. However, on statistically comparing the results at 168 days between the groups, significant improvements were only observed in pain levels (p = 0.02) and DUH (p=0.003).Conclusions. The injection of ADSVF plus fat micrografts is a reproducible, and safe technique. Pain and digital ulcers in the hands of patients with SS can be treated with this treatment. Trial Registration: Retrospectively registered in ClinicalTrials.gov with identifier NCT04387825

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