scholarly journals SAT0325 STELLATE BLOCKADE COMBINED TO ILOPROST AS SUPPORTIVE TREATMENT OPTION IMPROVES PAIN AND ISCHAEMIC SYMPTOMS IN PATIENTS WITH SYSTEMIC SCLEROSIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1108.2-1108
Author(s):  
F. Höcketstaller ◽  
U. Henkemeier ◽  
M. Zimmermann ◽  
H. Burkhardt ◽  
F. Behrens ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Combined Treatment ◽  
Common Symptom ◽  
Digital Ulcers ◽  
Research Support ◽  
Pronounced Increase ◽  
Capillary Microscopy ◽  
Patient Reported ◽  
The Impact ◽  
Subjective Sensation

Background:Peripheral ischaemia is a common symptom in systemic sclerosis (SSc) patients with risk of development of digital ulcers (DU). For its treatment, intravenous iloprost is the most effective option. Accompanying pain symptoms worsen the ischaemic symptoms, so a combination with anaesthetic procedures may improve ischaemic status and the subjective sensation of raynaud and pain. The aim of this study was to observe the impact of a combined treatment of iloprost with stellate blockade (ILOST) in improvement of ischaemic symptoms compared to iloprost treatment only (ILO).Objectives:To evaluate efficacy of the ILOST treatment on changes in vascularisation and sensation of patients with SSc and indication for vasodilatative treatment with Iloprost.Methods:Twenty SSc-patients with indication for ILO-treatment (prophylactic or due to digital ulcerations (DU)) will be included in a prospective observational study. Patients will be offered to combine ILO with stellate blockade (ILOST). Beside documentation of disease activity characteristics (mRSS, number of DU, capillary microscopy at baseline, after ILO-treatment and at week 12), patients are assessed using fluorescence-optical imaging (FOI) as innovative method for illustration of changes in microvascularisation and patient reported outcomes (DASH, VAS) at week 12.Results:This interims analysis includes the result of the first 11 patients treated. Mean baseline characteristics (age and gender) are well balanced. Iloprost treatment was initiated due to prophylactic treatment to avoid DU in all patients. 100% of the patients in the ILOST-group were diagnosed as limited SSc compared to 60% in the ILO-group (diffuse type with 40%). All patients showed abnormalities in capillary microscopy (ILOST group: 83,3% late pattern 16.7% active pattern; ILO group: 80% late pattern, 20% early pattern). MRSS was low in both groups with 1.8, the disease duration in mean 15.3 years in the ILOST-group compared to 13.2 in the ILO-group, respectively. In both groups, no new DU occurred in the 12-week follow-up. Improvement in VAS pain was reported in 83% of the patients in the ILOST group compared to 60% in the ILO group. DASH improved with a mean of 5.5 points in the ILOST group compared to 3 points in the ILO group. FOI was compared individual at both arms in the ILOST group only. The arm with stellatum blockade showed a pronounced increase of FOI signals of 5% in mean whereas the opposite site showed a decrease of the signal shortly after ILO treatment indicating a pronounced increase of vascularisation in the ILOST treated body site.Conclusion:A new treatment approach to improve acute ischaemic symptoms was tested by combining stellate blockade to iloprost treatment. No new DU occurred up to 12 weeks after treatment in all patients of both groups indicating the relevance of iloprost as effective vascular dilatative therapy in SSc. The additional intervention was well tolerated and asked to repeat. Subjective sensation on pain of the hands as well as DASH was improved in the combined group. FOI showed a relevant increase in vascularisation in the blockade arm compared to the opposite site in which signals decreased indicating a stronger effect of the combined treatment for improvement of vascularisation.Disclosure of Interests:Franziska Höcketstaller Grant/research support from: Rheumazentrum Rhein-Main, Ulf Henkemeier: None declared, Michael Zimmermann: None declared, Harald Burkhardt Grant/research support from: Pfizer, Roche, Abbvie, Consultant of: Sanofi, Pfizer, Roche, Abbvie, Boehringer Ingelheim, UCB, Eli Lilly, Chugai, Bristol Myer Scripps, Janssen, and Novartis, Speakers bureau: Sanofi, Pfizer, Roche, Abbvie, Boehringer Ingelheim, UCB, Eli Lilly, Chugai, Bristol Myer Scripps, Janssen, and Novartis, Frank Behrens Grant/research support from: Pfizer, Janssen, Chugai, Celgene, Lilly and Roche, Consultant of: Pfizer, AbbVie, Sanofi, Lilly, Novartis, Genzyme, Boehringer, Janssen, MSD, Celgene, Roche and Chugai, Ulrich Drott: None declared, Michaela Köhm Grant/research support from: Pfizer, Janssen, BMS, LEO, Consultant of: BMS, Pfizer, Speakers bureau: Pfizer, BMS, Janssen, Novartis

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 THU0340 DURATION AND SYSTEMIC SCLEROSIS SUBTYPE ARE ASSOCIATED WITH DIFFERENT GUT MICROBIOME PROFILES

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 401.2-401
Author(s):  
Y. Braun-Moscovici ◽  
S. Ben Simon ◽  
K. Dolnikov ◽  
S. Giryes ◽  
D. Markovits ◽  
...  
Keyword(s):  
Species Richness ◽  
Systemic Sclerosis ◽  
Gut Microbiota ◽  
Disease Duration ◽  
Alpha Diversity ◽  
Rrna Gene ◽  
Digital Ulcers ◽  
Diffuse Disease ◽  
Probiotic Treatment ◽  
The Impact

Background:A growing body of evidence suggests that the gut microbiota plays a significant role in the development of autoimmune diseases. Altered microbiota composition was associated with gastrointestinal and extraintestinal features in systemic sclerosis (SSc) patients.Objectives:To look for differences in gut microbiota between SSc patients regarding disease duration, disease subset and occurrence of digital ulcers (DU).Methods:SSc patients seen at our center were recruited in a prospective study. The exclusion criteria included antibiotic or probiotic treatment during the month prior to recruitment, recent hospitalization, BMI>30, diabetes mellitus or concomitant inflammatory bowel disease. Fecal samples were processed and 16S rRNA gene sequences were analyzed using the QIIME2 packageWeighted (quantitative) and unweighted (qualitative) UniFrac distances, alpha diversity for richness and homogeneity, taxa plots for species and phyla and ANCOM analyses were performed.Results:During July 2018-May 2019, 26 SSc patients (mean age [SD] 53[12.7] years) and disease duration 8.8 [7.1] years) fulfilled the criteria and were willing to participate in the study. Thirteen patients had diffuse SSc, 16 patients had active DU, 8 patients had Raynaud’s phenomenon only without DU, 2 patients had past DU. The microbiota was significantly more similar between patients without active DU compared to those with active DU (P=0.024), but species richness did not differ. Patients with SSc duration less than 6 years had significantly different microbiota compared to long-lasting SSc (unweighted PCoA – q=0.031). Significant variations concerning quantitative and qualitative UniFrac distances (q=0.063, q=0.005) and species richness (q=0.009) were found among patients with diffuse compared to limited SSc. Limited SSc was associated with greater species richness. Taxa plot analysis revealed higher relative abundance of Firmicutes in diffuse disease and of Actinobacteria and Bacteroidetes in limited SSc.Conclusion:Disease duration, disease subset and active DU were associated with shifts in the microbiome of SSc patients. The impact of these changes on disease progression needs further elucidation.Figure:Disclosure of Interests:Yolanda Braun-Moscovici: None declared, Shira Ben Simon: None declared, Katya Dolnikov: None declared, Sami Giryes: None declared, Doron Markovits: None declared, Yonit Tavor: None declared, Kohava Toledano: None declared, Alexandra Balbir-Gurman Consultant of: Novartis, Omry Koren: None declared

scholarly journals What narrative devices do people with systemic sclerosis use to describe the experience of pain from digital ulcers: a multicentre focus group study at UK scleroderma centres

BMJ Open ◽  
2020 ◽  
Vol 10 (6) ◽  
pp. e037568
Author(s):  
Jennifer Jones ◽  
Michael Hughes ◽  
John Pauling ◽  
Rachael Gooberman-Hill ◽  
Andrew J Moore
Keyword(s):  
Systemic Sclerosis ◽  
Focus Group ◽  
Focus Groups ◽  
Patient Experience ◽  
Patient Reported Outcome ◽  
Digital Ulcers ◽  
Group Setting ◽  
Focus Group Study ◽  
Psychological Reactions ◽  
Patient Reported

ObjectivesDigital ulcers (DUs) are a common complication in systemic sclerosis (SSc). No existing studies have specifically reported on the qualitative patient experience of DU pain, and our current patient-reported outcome measure (PROM) does not capture the multifaceted painful experience of SSc-DU. Our aim was to examine the patient experience of SSc-DU pain.DesignFocus groups with people diagnosed with SSc who had experienced DUs were conducted using a topic guide developed by people with SSc, experts in SSc and experienced qualitative researchers. Focus groups were continued until data saturation had been reached. The focus groups were audio recorded, transcribed verbatim, anonymised and analysed using inductive thematic analysis. Our current study is an integration of the data from these focus groups to specifically examine the patient experience of DU pain.SettingThree specialist scleroderma units across the UK (Bath, Manchester and London).ParticipantsFour focus groups were undertaken; 29 adults (20 women, 9 men) with SSc and a spectrum of historical DUs participated. We included participants with a diverse demographic (including ethnic) background and disease-related characteristics.ResultsFive narrative devices were identified, which encompass how people describe the pain from SSc-DUs: ‘Words to express DU-associated pain’, ‘Descriptions of physical and psychological reactions to pain’, ‘Comparisons with other painful events’, ‘Descriptions of factors that exacerbate pain’ and ‘Descriptions of strategies for coping with the pain’.ConclusionThe experience of SSc-DU pain leads to the use of graphic language and rich description by participants in the focus group setting. Existing SSc-DU outcomes do not adequately capture the patient experiences of SSc-DU pain. Our findings further highlight the multifaceted nature of SSc-DUs and will hopefully support the development of a novel PROM to assess the severity and impact of SSc-DUs.

scholarly journals POS0319 PERFORMANCE OF PATIENT REPORTED OUTCOMES (PROs) IN SCLERODERMA PATIENTS WITH REDUCED LUNG FUNCTION IN AN OBSERVATIONAL COHORT

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 387.1-387
Author(s):  
M. G. Lazzaroni ◽  
G. Abignano ◽  
M. Wilson ◽  
V. Kakkar ◽  
F. Del Galdo
Keyword(s):  
Lung Function ◽  
Patient Reported Outcomes ◽  
Hand Function ◽  
Health Assessment ◽  
Digital Ulcers ◽  
General Function ◽  
Research Support ◽  
Cross Sectional ◽  
Patient Reported ◽  
Spearman Test

Background:Patient Reported Outcomes (PROs) are used to capture disease impact on patients’ Health Related Quality of Life (HRQoL) and they have been increasingly used as endpoints in clinical trials for Systemic Sclerosis (SSc). The DeSScipher project within the EUSTAR group highlighted dyspnea as one of the factors more strongly related with the highest SHAQ scores (1). Nevertheless, the SENSCIS trial in SSc-ILD (2) showed the efficacy of Nintedanib in reducing the annual rate of FVC loss, as compared to placebo, without significant changes in the PROs used as secondary endpoints (St. George’s Respiratory Questionnaire and the FACIT-Dyspnoea questionnaire). Since patient’s perspective is a crucial determinant to define the overall relevance of an intervention, the performance of PROs in reflecting different lung functional stages is a relevant issue in SSc.Objectives:To analyse in a prospective SSc cohort the inference of reduced lung function as measured by FVC on median PRO scores and the correlation among distinct commonly used PROs.Methods:A cross-sectional study was performed on data exported from the STRIKE database regarding SSc patients followed in Leeds Scleroderma Programme for SSc. Data included records of periodical visits with scores of different PROs commonly used in SSc (Scleroderma-Health Assessment Questionnaire (SHAQ), Cochin Hand Function Scale (CHFS) and Borg dyspnoea scale) and FVC%-predicted (%pFVC) and DLCO%-predicted (%pDLCO). SHAQ score was calculated as the mean value of HAQ (0-3) with the average of the 7-VAS scores divided by 3.33. The 7-VAS (score 1-10) were 1: pain, 2: general function, 3: arthritis, 4: gastro-intestinal, 5: dyspnoea, 6: Raynaud Phenomena; 7: digital ulcers. The correlation of FVC with distinct PROs, and the inter-PRO correlation, were analysed through the non-parametric Spearman test.Results:Complete data were available from 182 visits of 87 SSc patients (41 with diffuse and 46 with limited cutaneous involvement). Mean %pFVC was 95.16 ±24.93 (median 95) and mean %pDLCO was 59.31±16.51 (median 59). Overall, FVC and DLCO showed a moderate correlation with SHAQ (r=-0.36, p<0.001 and r=-0.24, p:0.001 respectively), while Borg score showed a stronger negative correlation with FVC and DLCO (r:-0.42 and r-0.38, p<0.001 for both). In a sub-analysis of patients grouped by FVC, patients with FVC 50-70% showed a significant correlation of FVC with SHAQ (r =-0.47, p:0.012), which was not present in patients with FVC 70-90% (r:-0.23, p:0.13). VAS-5 dyspnoea and Borg were not associated with FVC in these two subgroups of patients.Inter PROs analysis showed that CHFS score had a stronger correlation with SHAQ than Borg dyspnoea score in the overall population (r:0.86 vs. r:0.57, both p<0.001).Conclusion:The analysis of a single centre prospective cohort of SSc patients, suggests a small inference of lung function in the overall SHAQ. The stronger correlation of SHAQ with CHFS, than with Borg score, suggests a higher weight of hand function on SHAQ in this population with relatively conserved lung function. In patients with %pFVC <70%, the correlation with SHAQ was stronger than in patients with %pFVC >70%, suggesting that mild reductions in FVC might not be perceived by the patients, or at least they might not modify HrQoL as measured by SHAQ.References:[1]Jaeger VK, et al. Rheumatology 2017;57(3):441-50.[2]Distler O, et al. N Engl J Med 2019; 380:2518-2528.Disclosure of Interests:Maria Grazia Lazzaroni Grant/research support from: Boehringer-Ingelheim, Giuseppina Abignano: None declared, Michelle Wilson: None declared, Vishal Kakkar: None declared, Francesco Del Galdo Consultant of: Astra-Zeneca, Boehringer-Ingelheim, Mitsubishi-Tanabe, Capella Biosciences, Kymab, Actelion, Grant/research support from: Astra-Zeneca, Boehringer-Ingelheim, Mitsubishi-Tanabe, Capella Biosciences, Kymab, Actelion

scholarly journals THU0498 PATIENT-REPORTED TREATMENT BURDEN AND ITS IMPACT ON QUALITY OF LIFE IN JUVENILE IDIOPATHIC ARTHRITIS: RESULTS FROM THE PHARMACHILD REGISTRY

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 486-487
Author(s):  
A. Alongi ◽  
A. Consolaro ◽  
G. Vijatov-Djuric ◽  
G. Filocamo ◽  
O. Vougiouka ◽  
...  
Keyword(s):  
Medication Adherence ◽  
Disease Activity ◽  
Patient Reported Outcomes ◽  
Sleep Problems ◽  
Psychosocial Health ◽  
Research Support ◽  
School Problems ◽  
Patient Reported ◽  
The Impact

Background:Juvenile Idiopathic Arthritis (JIA) patients experience impaired health and wellbeing due to multiple causes of physical and psychosocial distress, including treatment burden. Despite emerging evidence of its relevance [1], the contribution of treatment adverse events to patient-reported outcomes (PROs) in JIA has been poorly explored.Objectives:To evaluate and rank the impact of patient-reported adverse events (AEs) on overall wellbeing, health-related quality of life (HRQoL), school problems and self-reported medication adherence using data from Pharmachild, a large international JIA pharmacovigilance registry.Methods:Registry entries on 5340 prospective visits of 2251 patients enrolled till December 2018 were analyzed; all included patients were treated with at least one DMARDS or Biologic agent at the time of visit. In the Juvenile Arthritis Multidimensional Assessment Report (JAMAR), patients and parents compiled a checklist of treatments, side effects, self-reported adherence, administration difficulties and disease-related school problems occurred in the previous 4 weeks. Evaluated outcomes included patient acceptable symptom state (PASS), VAS-measured patient assessment of overall wellbeing (PGA) and HRQoL, assessed through the physical health (PhH) and psychosocial health (PsH) subscales. The relationships between AEs and PROs were tested through generalized linear models, accounting for disease activity and symptoms levels. Bayesian Networks were used to explore the causal effects of specific AEs on outcomes to disentangle the confounding role of disease status.Results:AEs were reported in 22.9% of visits. For similar levels of physician global assessment (MD global), patient-assessed disease activity, pain and function, patients reporting AEs had worse PGA, PsH, and lower probability of reaching PASS (fig. 1, all p-values <0.001). The impact of AEs on PGA was small but not trivial (effect size η20.031) and appears to be mediated by effects on PsH and school problems (p <0.001). Non-linear regression modelling revealed a significant moderating effect of MD global levels < 2.5 on the relationship between AEs and PGA (p 0.003), indicating that the impact of AEs is higher for lower disease activity states. AEs predicted self-reported medication adherence (p<0.001), even when adjusted for the number of administered treatments. In the Bayesian network model, mood swing and sleep problems emerged as the most influential items affecting PsH, (respectively, total effect 2.62 and 1.25, both p< 0.001). Fig. 2 shows the total standardized effect of specific AEs on mean PsH levels. Nausea had the highest impact on treatment adherence (total effect -0.0541, p <0.001), being the only AE directly linked to drug refusal.Conclusion:AEs have a measurable effect on the wellbeing and psychosocial health of JIA patients, particularly when disease activity is low, and significantly affect school activity and medication adherence. Mood swings and sleep problems show the strongest influence on HRQoL. Addressing AEs appears important to reduce disease impact, improve patients’ satisfaction and therapeutic compliance.References: :[1]Weitzman, Elissa R., et al. Journal of patient-reported outcomes 2.1 (2018): 1.Acknowledgments:for the Paediatric Rheumatology International Trials Organisation (PRINTO)Disclosure of Interests: :Alessandra Alongi: None declared, Alessandro Consolaro Grant/research support from: Pfizer Inc., AlfaSigma, Speakers bureau: AbbVie, Gordana Vijatov-Djuric: None declared, Giovanni Filocamo: None declared, Olga Vougiouka: None declared, Alma Nunzia Olivieri: None declared, Cristina Herrera Mora: None declared, Wolfgang Emminger: None declared, Angelo Ravelli: None declared, Nicolino Ruperto Grant/research support from: Bristol-Myers Squibb, Eli Lily, F Hoffmann-La Roche, GlaxoSmithKline, Janssen, Novartis, Pfizer, Sobi (paid to institution), Consultant of: Ablynx, AbbVie, AstraZeneca-Medimmune, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lily, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, Sanofi, Servier, Sinergie, Sobi, Takeda, Speakers bureau: Ablynx, AbbVie, AstraZeneca-Medimmune, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lily, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, Sanofi, Servier, Sinergie, Sobi, Takeda

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 Ischemic Digital Ulcers Affect Hand Disability and Pain in Systemic Sclerosis

2014 ◽  
Vol 41 (7) ◽  
pp. 1317-1323 ◽  
Author(s):  
Luc Mouthon ◽  
Patrick H. Carpentier ◽  
Catherine Lok ◽  
Pierre Clerson ◽  
Virginie Gressin ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Hand Function ◽  
Time Interval ◽  
Digital Ulcers ◽  
Raynaud Phenomenon ◽  
Hand Disability ◽  
The Mean ◽  
Noninterventional Study ◽  
The Impact ◽  
Mean Time

Objective.Ischemic digital ulcers (DU) are frequent and severe complications of systemic sclerosis (SSc). The purpose of our study was to assess the effect of DU on hand disability and pain in patients with SSc.Methods.The Evaluation of the Impact of Recurrent Ischemic DU on Hand Disability in Patients with SSc (ECLIPSE) is a prospective, multicenter, noninterventional study with a 2-year followup. Patients with SSc who experienced at least 1 DU in the previous year and received bosentan therapy were included between October 2009 and March 2011. This cohort is described at the time of inclusion.Results.There were 190 patients (132 females) from 53 centers. Mean age ± SD was 43 ± 15 years at SSc diagnosis and 53 ± 15 years at inclusion. In 105 patients (56.2%), DU were the first non-Raynaud symptoms of SSc. The mean time interval between the occurrence of Raynaud phenomenon and the first DU episode was 6.6 ± 9.1 years. The mean numbers of active DU and fingers affected per patient for both hands were 2.3 ± 1.8 and 2.2 ± 1.6, respectively. Presence of active DU at inclusion was significantly associated with pain and impaired hand function: Visual Analog Scale for pain (0 to 10) was 6.2 ± 2.6 versus 2.5 ± 2.4 (p < 0.0001) and Cochin Hand Function Scale for hand disability (0 to 90) was 38 ± 20 versus 25 ± 19 (p < 0.0001), respectively.Conclusion.DU represent a major sign of SSc, often affecting multiple fingers and both hands. They are significantly associated with pain and hand disability.

scholarly journals Thermographic Abnormalities are Associated with Future Digital Ulcers and Death in Patients with Systemic Sclerosis

2016 ◽  
Vol 43 (8) ◽  
pp. 1519-1522 ◽  
Author(s):  
Michael Hughes ◽  
Jack Wilkinson ◽  
Tonia Moore ◽  
Joanne Manning ◽  
Paul New ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Vascular Disease ◽  
Disease Severity ◽  
Case Note ◽  
Digital Ulcers ◽  
Retrospective Case ◽  
Patient Reported ◽  
Multiple Episodes ◽  
Case Note Review

Objective.Capillaroscopic abnormalities are predictive of future digital ulcers (DU). Our aim was to investigate whether functional digital vascular disease (thermographically assessed) is also associated with future DU.Methods.A retrospective case note review of patients with systemic sclerosis (SSc) undergoing thermography and who were followed for up to about 3 years.Results.There were 138 patients (equal mixture of normal/abnormal thermography). Patients with abnormal thermography were more likely to develop DU (clinician-observed and/or patient-reported, OR 2.84, p = 0.021), including multiple episodes, and more likely to die (OR 5.42, p = 0.050).Conclusion.Abnormal thermography is associated with DU and disease severity in patients with SSc.

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