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Reduced Health-Related Quality of Life in Patients With Systemic Sclerosis: A Cross-Sectional Analysis of PROMIS Global Health Data From the International COVAD-2 e-Survey
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Vaccine hesitancy is considered a major barrier to achieving herd immunity against COVID-19. While multiple alternative and synergistic approaches including heterologous vaccination, booster doses, and antiviral drugs have been developed, equitable vaccine uptake remains the foremost strategy to manage pandemic. Although none of the currently approved vaccines are live-attenuated, several reports of disease flares, waning protection, and acute-onset syndromes have emerged as short-term adverse events after vaccination. Hence, scientific literature falls short when discussing potential long-term effects in vulnerable cohorts. The COVAD-2 survey follows on from the baseline COVAD-1 survey with the aim to collect patient-reported data on the long-term safety and tolerability of COVID-19 vaccines in immune modulation. The e-survey has been extensively pilot-tested and validated with translations into multiple languages. Anticipated results will help improve vaccination efforts and reduce the imminent risks of COVID-19 infection, especially in understudied vulnerable groups.
Background
Systemic sclerosis (SSc) is a rare autoimmune disease characterized by fibrosis of the skin and the involvement of multiple internal organs. Previous studies reported poorer health-related quality of life (HRQoL) in patients with SSc compared with the general population. However, very little is known about how HRQoL in SSc patients compares with that in patients with other systemic autoimmune diseases, such as rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), and Sjogren’s syndrome (SjS). Thus, the main aim of this study was to compare HRQoL in SSc patients, patients with other rheumatic diseases, and the general population.
Methods
In this cross-sectional study, patients from the rheumatology clinics of Seoul National University Hospital with SSc, RA, SLE, and SjS were enrolled via a random sampling technique. HRQoL was captured by the Short Form (36) health survey (SF-36), the Short Form Six-Dimensional health index (SF-6D), and the EuroQol Five-Dimensional descriptive system (EQ-5D). Demographic characteristics and standardized disease activity for each disease were also obtained. Previously reported data from 600 healthy Koreans were used for the healthy controls. An ANCOVA test was used to compare the SF-36, SF-6D, and EQ-5D values between study subjects with adjustments for age, sex, disease duration, comorbidities, and disease activity status.
Results
One hundred twenty patients were included in each of the SSc, RA, SLE, and SjS cohorts. Patients with rheumatic diseases had significantly lower SF-36, SF-6D, and EQ-5D scores than healthy controls (all P < 0.001). After statistical adjustments, SSc patients reported significantly lower mental component summary (MCS) scores than patients with RA (P < 0.001) or SLE (P = 0.001). Specifically, the mental health and general health domains were significantly lower in SSc patients than reported in RA or SLE patients (P < 0.001 and P = 0.001, respectively, in both domains). In SSc patients, higher modified Rodnan skin scores (mRSS) correlated with lower MCS scores.
Conclusions
SSc patients report poorer HRQoL than patients with RA or SLE. The extent of skin involvement is associated with poorer HRQoL in SSc patients.
Trial registration
NCT03257878. Registered 22 August 2017
Electronic supplementary material
The online version of this article (10.1186/s13075-019-1842-x) contains supplementary material, which is available to authorized users.
Background:
Absolute nailfold capillary number should be a putative biomarker in selected rheumatic diseases but could be time consuming and not highly repeatable.
Objective:
To validate an automated software for absolute nailfold capillary number and density evaluation, on nailfold videocapillaroscopy (NVC) images in systemic sclerosis (SSc).
Methods:
An automated software to count nailfold capillary number (AUTOCAPI) had been constructed, through an exploratory image set. Subsequently, application rules have been created to define the region of interest (ROI) in NVC images, through a training images set. The software reliability was assessed through calculation of the intraclass correlation coefficient (ICC) between automatic and manual counting, by four independent observers, on the same NVC images.
Results:
The following ICC's were obtained per observer, for the SSc patients (40 images), the healthy (20 images) and the primary Raynaud's (PRP) subgroups (20 images) respectively: 0.94, 0.81, 0.62 (observer 1); 0.94, 0.91, 0.67 (observer 2); 0.88, 0.56, 0.64 (observer 3) and 0.88, 0.85, 0.85 (observer 4).
Conclusion:
The validation of an automated software for measuring absolute nailfold capillary number and density in SSc was achieved. The integration into the pre-existing imaging software should make the assessment of the capillary number in NVC, easier, quicker and standardized. This article is protected by copyright. All rights reserved.
The use of global health items permits an efficient way of gathering general perceptions of health. These items provide useful summary information about health and are predictive of health care utilization and subsequent mortality.
Analyses of 10 self-reported global health items obtained from an internet survey as part of the Patient-Reported Outcome Measurement Information System (PROMIS) project. We derived summary scores from the global health items. We estimated the associations of the summary scores with the EQ-5D index score and the PROMIS physical function, pain, fatigue, emotional distress, and social health domain scores.
Exploratory and confirmatory factor analyses supported a two-factor model. Global physical health (GPH; 4 items on overall physical health, physical function, pain, and fatigue) and global mental health (GMH; 4 items on quality of life, mental health, satisfaction with social activities, and emotional problems) scales were created. The scales had internal consistency reliability coefficients of 0.81 and 0.86, respectively. GPH correlated more strongly with the EQ-5D than did GMH (r = 0.76 vs. 0.59). GPH correlated most strongly with pain impact (r = -0.75) whereas GMH correlated most strongly with depressive symptoms (r = -0.71).
Two dimensions representing physical and mental health underlie the global health items in PROMIS. These global health scales can be used to efficiently summarize physical and mental health in patient-reported outcome studies.
Systemic sclerosis, also known as scleroderma, is a rare and complex autoimmune connective-tissue disease. Once considered an untreatable and unpredictable condition, research advancements have improved our understanding of its disease pathogenesis and clinical phenotypes and expanded our treatment armamentarium. Early and accurate diagnosis is essential, while ongoing efforts to risk stratify patients have a central role in predicting both organ involvement and disease progression. A holistic approach is required when choosing the optimal therapeutic strategy, balancing the side-effect profile with efficacy and tailoring the treatment according to the goals of care of the patient. This Seminar reviews the multiple clinical dimensions of systemic sclerosis, beginning at a precursor very early stage of disease, with a focus on timely early detection of organ involvement. This Seminar also summarises management considerations according to the pathological hallmarks of systemic sclerosis (eg, inflammation, fibrosis, and vasculopathy) and highlights unmet needs and opportunities for future research and discovery.
Objectives:
Racial factors play a significant role in SSc. We evaluated differences in SSc presentations between white patients (WP), Asian patients (AP) and black patients (BP) and analysed the effects of geographical locations.
Methods:
SSc characteristics of patients from the EUSTAR cohort were cross-sectionally compared across racial groups using survival and multiple logistic regression analyses.
Results:
The study included 9162 WP, 341 AP and 181 BP. AP developed the first non-RP feature faster than WP but slower than BP. AP were less frequently anti-centromere (ACA; odds ratio (OR) = 0.4, P < 0.001) and more frequently anti-topoisomerase-I autoantibodies (ATA) positive (OR = 1.2, P = 0.068), while BP were less likely to be ACA and ATA positive than were WP [OR(ACA) = 0.3, P < 0.001; OR(ATA) = 0.5, P = 0.020]. AP had less often (OR = 0.7, P = 0.06) and BP more often (OR = 2.7, P < 0.001) diffuse skin involvement than had WP. AP and BP were more likely to have pulmonary hypertension [OR(AP) = 2.6, P < 0.001; OR(BP) = 2.7, P = 0.03 vs WP] and a reduced forced vital capacity [OR(AP) = 2.5, P < 0.001; OR(BP) = 2.4, P < 0.004] than were WP. AP more often had an impaired diffusing capacity of the lung than had BP and WP [OR(AP vs BP) = 1.9, P = 0.038; OR(AP vs WP) = 2.4, P < 0.001]. After RP onset, AP and BP had a higher hazard to die than had WP [hazard ratio (HR) (AP) = 1.6, P = 0.011; HR(BP) = 2.1, P < 0.001].
Conclusion:
Compared with WP, and mostly independent of geographical location, AP have a faster and earlier disease onset with high prevalences of ATA, pulmonary hypertension and forced vital capacity impairment and higher mortality. BP had the fastest disease onset, a high prevalence of diffuse skin involvement and nominally the highest mortality.
Objectives:
PROMIS-29 is a generic health-related quality of life instrument. Our objective was to assess the reliability, construct validity, and responsiveness to change of PROMIS-29 in systemic sclerosis-associated interstitial lung disease (SSc-ILD).
Methods:
Seventy-three participants with SSc-ILD were administered patient reported outcomes (PROs) at baseline and follow-up visits which included PROMIS-29 and other measures of generic health, dyspnea, and cough instruments. We assessed internal consistency reliability using Cronbach's α, an alpha of ≥ 0.70 was considered satisfactory. We assessed the responsiveness to change using linear regression models.
Results:
Mean age of the participants was 51.9 years and the mean disease duration was 7.9 years after first non-Raynaud's symptom. Of the 73 participants, 56.2% were classified as diffuse SSc and 26% limited SSc. The baseline (mean ± SD) FVC % predicted was 73.9±15.5 with a DLCO % predicted of 57.7±21.1; 95.9% had fibrotic NSIP pattern on HRCT. PROMIS-29 scores were 0.2 to 0.9 SD below the US population. Cronbach's α reliability was acceptable for all domains (ranged from 0.77 to 0.98). All scales showed statistically significant correlations with hypothesised PROMIS-29 domains (p≤0.05 for all comparisons). PROMIS-29 showed none-to-small discriminatory ability in comparison with physiologic measures (FVC and DLCO). There was no significant relationship between the change in FVC versus the change in PROMIS-29 measures over time.
Conclusions:
PROMIS-29 has adequate reliability and construct validity for evaluation in SSc-ILD. It has moderate-to-large correlations with other PROs. The PROMIS-29 domains were not found to change over time in this cohort, likely due to stable nature of the observational cohort.
Objectives:
The purpose of this study was to assess health-related quality of life (HRQoL) and disease perception in a large, international group of patients with systemic sclerosis (SSc).
Methods:
We placed a standardized questionnaire on a website for patient access. Socio-demographic information, disease characteristics, and self-assessment questionnaires-the Short Form 36 (SF-36) and the Revised Illness Perception Questionnaire (IPQ-R)-were collected.
Results:
A total of 1902 patients from 60 countries were included. HRQoL appeared to be impaired in SSc, particularly for physical health (PCS, mean ± SD = 43.4 ± 23.4). SSc patients also had strong perceptions about the chronic nature and negative consequence of the disease, and experienced negative emotions due to SSc. Patients with diffuse cutaneous SSc had a poorer HRQoL than those with limited cutaneous SSc, for both physical (PCS, mean ± SD = 46.6 ± 23.7 vs. 39.8 ± 22.3; p < 0.0001) and mental components (MCS, mean ± SD = 53.8 ± 23.0 vs. 50.3 ± 23.2; p = 0.003). Late-stage SSc patients were more likely to perceive their disease chronic (p < 0.0001), less controllable (p = 0.03) and with more consequences (p = 0.008), but they had a better understanding of their disease and experienced fewer negative emotions. Raynaud's phenomenon and gastrointestinal complications were the organ involvements with the greatest impact on QoL, they were the two variables associated with the most negative perception of illness severity.
Conclusion:
This study, performed on the largest group ever set up for this purpose, confirms the major impact on QoL and the negative perceptions of their disease expressed by SSc patients. However, the perception of this illness tended to improve with disease duration, suggesting that patients find effective coping strategies.
The health assessment questionnaire (HAQ), a self administered instrument to determine physical disability, was completed by 211 patients with systemic sclerosis who subsequently received scleroderma examinations. The mean HAQ disability index for the entire group was 0.92. Patients with high skin scores had significantly higher disability indices as compared to patients with low skin scores (p < 0.001). Higher disability indices were found for patients with joint pain, tendon rubs, and contractures. However, the presence of ulcers on the digital tip did not interfere with patients' abilities to function as measured by this scale. Intensive hand evaluations were performed on a subset of these 211 patients with systemic sclerosis (n = 80). Grip strength, thumb abduction, wrist extension, and motion of the index and middle fingers significantly correlated with the HAQ scores (p < 0.01). The findings from this study suggest that patients with systemic sclerosis have significant physical disability. Furthermore, the HAQ appears to be useful in assisting health professionals in quantitating this disability.
Patient-reported outcomes (PROs) are essential when evaluating many new treatments in health care; yet, current measures have been limited by a lack of precision, standardization, and comparability of scores across studies and diseases. The Patient-Reported Outcomes Measurement Information System (PROMIS) provides item banks that offer the potential for efficient (minimizes item number without compromising reliability), flexible (enables optional use of interchangeable items), and precise (has minimal error in estimate) measurement of commonly studied PROs. We report results from the first large-scale testing of PROMIS items.
Fourteen item pools were tested in the U.S. general population and clinical groups using an online panel and clinic recruitment. A scale-setting subsample was created reflecting demographics proportional to the 2000 U.S. census.
Using item-response theory (graded response model), 11 item banks were calibrated on a sample of 21,133, measuring components of self-reported physical, mental, and social health, along with a 10-item Global Health Scale. Short forms from each bank were developed and compared with the overall bank and with other well-validated and widely accepted ("legacy") measures. All item banks demonstrated good reliability across most of the score distributions. Construct validity was supported by moderate to strong correlations with legacy measures.
PROMIS item banks and their short forms provide evidence that they are reliable and precise measures of generic symptoms and functional reports comparable to legacy instruments. Further testing will continue to validate and test PROMIS items and banks in diverse clinical populations.
Studies of systemic sclerosis (SSc) have enlisted measures of physical function and generic health-related quality of life in order to determine health status. However, the measurements obtained may not discriminate other essential quality of life (QOL) domains important to patients with SSc. Our study used qualitative methods to evaluate patients' assessment of disease and symptom burden in SSc.
We conducted 3 focus groups and 5 in-depth interviews of patients with SSc. Guiding questions were based on 5 themes: patient awareness, SSc-related problems, disease activity and progression, symptoms, and expectations. Thematic analysis was conducted using qualitative, grounded theory methodologies.
Symptoms such as pain (localized or generalized), fatigue, and malaise were reported to have major influence on daily activities and QOL. Gastrointestinal symptoms were among the worst prevalent and disruptive physical problems. All participants reported significant disruptions in their social lives, a burden considered by many as the worst consequence of their disease. All expressed major effects on their overall well-being because of emotional distress, including depression, low self-esteem, concerns with physical appearance, and uncertainty about future outcomes.
Patients with SSc report significant symptomatic and emotional burdens, which, in turn, affect their QOL and psychological well-being. Additional research and fuller awareness of the disease and symptom-related burdens experienced by patients with SSc may lead to additional relevant outcome measures and more effective overall treatment programs.
To assess the prevalence, course, and predictors of depression in patients with systemic sclerosis (SSc).
We conducted a comprehensive search in November 2006 of MEDLINE, PsycINFO, and CINAHL databases to identify original research studies published in any language that used a structured interview or validated questionnaire to assess major depressive disorder or clinically significant symptoms of depression in patients with SSc. The search was augmented by hand searching 26 selected journals through December 2006 and references from identified articles and reviews. Studies were excluded if only an abstract was provided or if depression was not measured by a validated method.
No studies used a structured clinical interview to assess the prevalence of major depressive disorder. The prevalence of clinically significant depressive symptoms was 51-65% based on 2 studies that used a Beck Depression Inventory (BDI) score >or=10 and 46-56% based on 2 studies that used a BDI score >or=11. These rates and those reported in 4 other studies that used different assessment tools (36-43%) were consistently high compared with other medical patient groups assessed with the same instruments and cutoffs. Methodologic issues limited the ability to draw strong conclusions from studies of predictors.
Symptoms of depression are common among patients with SSc. The high rates reported across studies suggest that routine screening is recommended. There is a need for studies that examine depression at different time points from the diagnosis of SSc and that systematically investigate factors associated with high levels of depressive symptoms.
To develop a provisional core set of response measures for clinical trials of systemic sclerosis (SSc).
The Scleroderma Clinical Trials Consortium (SCTC) conducted a structured, 3-round Delphi exercise to reach consensus on a core set of measures for clinical trials of SSc. Round 1 asked the SCTC investigators to list items in 11 pre-defined domains (skin, musculoskeletal, cardiac, pulmonary, cardio-pulmonary, gastrointestinal, renal, Raynaud phenomenon and digital ulcers, health-related quality of life and function, global health, and biomarkers) for SSc clinical trials. Round 2 asked respondents to rate the importance of the chosen items and was followed by a meeting, during which the Steering Committee discussed the feasibility, reliability, redundancy and validity of the items. Round 3 sought to obtain broader consensus on the core set measures. Members also voted on items that had data on feasibility but lacked data on reliability and validity, but may still be useful research outcome measures for future trials.
A total of 50 SCTC investigators participated in round 1, providing 212 unique items for the 11 domains. In all, 46 (92%) participants responded in round 2 and rated 177 items. The ratings of 177 items were reviewed by the Steering Committee and 31 items from the 11 domains were judged to be appropriate for inclusion in a 1-year multi-centre clinical trial. In total, 40 SCTC investigators completed round 3 and ranked 30 of 31 items as acceptable for inclusion in the core set. The Steering Committee also proposed 14 items for a research agenda.
Using a Delphi exercise, we have developed a provisional core set of measures for assessment of disease activity and severity in clinical trials of SSc.