Development of a new patient-reported outcome measure to assess activities and participation in people with systemic sclerosis: the Cochin 17-item Scleroderma Functional scale.

BACKGROUND: Patient-reported outcome measures (PROMs) aimed at assessing people with systemic sclerosis (SSc) have rarely involved the target population in the item- and domain-generation stage of the instrument construction. OBJECTIVES: To develop a new PROM assessing activities and participation in people with SSc. METHODS: A provisional International Classification of Functioning, Disability and Health (ICF)-based 65-item questionnaire previously developed from interviews of people with SSc was sent by email to all patients followed in the internal medicine department of Cochin hospital (n = 184) and enrolled in the Scleroderma Patient-centered Intervention Network Cohort. Items were reduced according to their metric properties. Dimensional structure of the questionnaire was assessed by principal component analysis, convergent and divergent validities by Spearman's rank correlation coefficient, internal consistency by Cronbach's α, and reliability by a test-retest method using the intraclass correlation coefficient (ICC) and Bland-Altman analysis. RESULTS: Overall, 113 of 184 patients (61·4%) completed the provisional questionnaire. The item-reduction process resulted in a 17-item questionnaire, the Cochin 17-item Scleroderma Functional scale (CSF-17). Principal component analysis extracted two dimensions: 10 items related to mobility (CSF-17 section A) and seven items related to general tasks (CSF-17 section B). We observed convergent validity of the CSF-17 total score with global activity limitation, pain, depression and aesthetic burden, and divergent validity with anxiety. Cronbach's α was 0·94 for section A and 0·95 for section B. ICC (n = 25 patients) was 0·92 for the CSF-17 total score. Bland-Altman analysis did not reveal a systematic trend for the test-retest. CONCLUSIONS: The CSF-17 is a new PROM assessing activities and participation specifically in people with SSc. Its content and construct validities are very high. What is already known about this topic? In the earliest stages of construction patient-reported outcomes (PROMs) for people with systemic sclerosis (SSc) rarely involve the target population. Instruments able to capture the specific needs of people with SSc in terms of activities and participation are lacking. What does this study add? The Cochin 17-item Scleroderma Functional Scale (CSF-17) is a new PROM assessing global activities and participation specifically in people with SSc. Patients' perspectives were prioritized at all stages of construction. What are the clinical implications of this work? The CSF-17 could be used in clinical practice and research to assess the efficacy of complex multidisciplinary interventions targeting activity limitations and participation restriction in people with SSc. Linked Comment: Clark and Denton. Br J Dermatol 2020; 183:610.

Non-medical cannabis in North America: an overview of regulatory approaches.

OBJECTIVES: The objective of this study was to describe existing regulations of non-medical cannabis legalization in North America to inform recommendations for future health policy. These regulations are among the first in the world and will set a precedent for other jurisdictions globally who legalize cannabis. STUDY DESIGN: This was a review of online grey literature on regulatory approaches to non-medical cannabis legalization in North American jurisdictions. METHODS: We conducted an internet search in June 2019 to identify government and public health resources published after January 1, 2012. We were able to achieve data saturation using a limited number of resources. Data extraction was conducted by two independent reviewers, with disagreements resolved by consensus. RESULTS: Eleven US states, the District of Columbia, and Canada have enacted legal recreational cannabis regulations. The legal age of cannabis possession matches the legal drinking age in all jurisdictions except one. Most consumption is in private residences only, with some provinces/territories permitting public consumption where tobacco is permitted. Most jurisdictions allow for home growing of up to 6 (US) or 4 (Canada) plants and a maximum possession of 1 oz. (US) or 1.06 oz. (Canada). Cannabis is available for purchase only in private retail stores in US states, while Canada has also legalized online sales. Impaired driving assessment is not cannabis-specific in most US states, while Canada has federal driving limits. CONCLUSIONS: Although North American approaches to regulating recreational cannabis use are consistent in many aspects, some exceptions exist (e.g., home growing, personal possession). More research is needed to assess the impact of variations in regulatory policies on potential harms from legalization to inform future policy decisions in North America and abroad. Complementary public health interventions will be crucial in ensuring public health and safety.

Are patient education materials about cancer screening more effective when co-created with patients? A qualitative interview study and randomized controlled trial.

Background: Patient education materials (pems) are frequently used to help patients make cancer screening decisions. However, because pems are typically developed by experts, they might inadequately address patient barriers to screening. We co-created, with patients, a prostate cancer (pca) screening pem, and we compared how the co-created pem and a pem developed by experts affected decisional conflict and screening intention in patients. Methods: We identified and used patient barriers to pca screening to co-create a pca screening pem with patients, clinicians, and researchers. We then conducted a parallel-group randomized controlled trial with men 40 years of age and older in Ontario to compare decisional conflict and intention about pca screening after those men had viewed the co-created pem (intervention) or an expert-created pem (control). Participants were randomized using dynamic block randomization, and the study team was blinded to the allocation. Results: Of 287 participants randomized to exposure to the co-created pem, 230 were analyzed, and of 287 randomized to exposure to the expert-created pem, 223 were analyzed. After pem exposure, intervention and control participants did not differ significantly in Decisional Conflict Scale scores [mean difference: 0.37 ± 1.23; 95% confidence interval (ci): -2.05 to 2.79]; in sure (Sure of myself, Understand information, Risk-benefit ratio, or Encouragement) scores (odds ratio: 0.75; 95% ci: 0.52 to 1.08); or in screening intention (mean difference: 0.09 ± 0.08; 95% ci: -0.06 to 0.24]). Conclusions: The effectiveness of the co-created pem did not differ from that of the pem developed by experts. Thus, pem developers should choose the method that best fits their goals and resources.

Frequency and impact of disease symptoms experienced by patients with systemic sclerosis from five European countries.

OBJECTIVES: Knowledge about the nature and impact of symptoms faced by patients with systemic sclerosis (SSc) is needed to identify targets for research and treatment. The aim of this study was to assess and compare the frequency and impact on everyday activities of SSc symptoms among patients from five European countries. METHODS: European patients with SSc were invited through announcements by patient associations to complete an online survey. The survey included items assessing the frequency of 40 SSc symptoms and the impact on daily activities, if present. Chi-square tests were utilised to assess the differences in frequency and impact of symptoms across countries. RESULTS: In total, 537 patients were included from France (n=111), the Netherlands (n=229), Spain (n=61), Switzerland (n=50), and the United Kingdom (n=86). Symptoms experienced by ≥ 70% of patients in all countries were fatigue, Raynaud's phenomenon, joint pain, and muscle pain. Twenty symptoms were experienced by ≥ 50% of patients in all countries. Thirty symptoms had an impact on daily activities in ≥ 50% of patients who reported that the symptom was present in all countries. There were significant differences among countries in the prevalence of 17 out of 40 symptoms. Furthermore, in 24 out of 40 symptoms significant differences in the proportion of patients reporting impact of a specific symptom on everyday activities were observed. CONCLUSIONS: European patients with SSc experience a broad range of symptoms that have an impact on everyday activities. International research initiatives should target common SSc symptoms cooperatively. Further research is needed to better understand the differences in SSc symptoms among countries.

Sex and time to diagnosis in systemic sclerosis: an updated analysis of 1,129 patients from the Canadian scleroderma research group registry.

OBJECTIVES: A previous study found that time to diagnosis was significantly longer from onset of Raynaud's phenomenon for women compared to men with diffuse systemic sclerosis (SSc) and that, in limited SSc, it was more than twice as long for women than men. That study was limited, however, by the small number of men in disease subtype subgroups. The objective of the present study was to investigate the association of sex with time to diagnosis of SSc using a substantially larger patient sample. METHODS: Association between sex and time to diagnosis was assessed overall and stratified based on diffuse versus limited disease using Kaplan-Meier curves and Cox proportional hazards models. RESULTS: There were 1,129 patients in the study (median age=56.0 years; 978 [86.6%] women). Time to diagnosis was significantly longer for women (median=1.1 years) than men (median 0.8= years; p=0.037) with diffuse SSc following onset of Raynaud's phenomenon. There were no significant or substantive sex differences in time to diagnosis after Raynaud's onset in limited SSc or from onset of first non-Raynaud's disease manifestation in diffuse or limited SSc. CONCLUSIONS: Time to diagnosis was significantly longer for women compared to men with diffuse SSc following onset of Raynaud's phenomenon, but the difference was small and unlikely to be clinically significant. There were no differences in time to diagnosis following Raynaud's onset in limited disease or following onset of first non-Raynaud's disease manifestation in diffuse or limited disease. Overall, sex does not appear to influence time to diagnosis meaningfully.

[Is there evidence that depression screening benefits patients with medical illness?: not yet]. / Depressionsscreening bei Patienten mit somatischen Erkrankungen: Der Nachweis des Nutzens steht noch aus.

Depression is increasingly recognized as a common and disabling condition, and improving depression care has been prioritized by policy-makers in Germany and elsewhere. Screening for depression, which refers to the routine use of depression screening tools or sets of questions to identify patients who may have depression, but whose depression is otherwise not recognized by their physician, is one solution that has been proposed. However, there has never been a clinical trial that has found better depression outcomes for patients screened for depression compared to patients not screened when the same treatment and care resources are made available to both groups. There are, on the other hand, many negative trials that have failed to find benefit to patients from depression screening. Thus, depression screening has not been shown to benefit patients, may lead to important harm, and would consume a substantial amount of scarce resources. Prior to recommending that physicians routinely screen patients for depression, well-designed randomized controlled trials are needed that compare depression outcomes for patients screened versus patients not screened with the same treatment options available to both screened and non-screened patients.

A systematic comparison of fatigue levels in systemic sclerosis with general population, cancer and rheumatic disease samples.

OBJECTIVES: There are no studies of fatigue levels in patients with SSc. The objective of this study was to compare fatigue in SSc to general population samples and patients with rheumatic diseases and cancer, where fatigue has been researched extensively. METHODS: SSc patients completed the General Fatigue Index (GFI) of the Multidimensional Fatigue Inventory. A systematic review was conducted to select comparison samples. Mean GFI scores from SSc patients were compared with mean scores from comparison samples with t-tests and Bonferroni corrections (family-wise P < 0.05). RESULTS: A total of 106 SSc patients were sampled (97 females; 28 diffuse SSc; 11.9 +/- 7.9 yrs since diagnosis). Based on comparisons from the systematic review, mean GFI scores in SSc (13.3 +/- 4.6) were significantly higher (greater fatigue; P < 0.05) than in two large population samples (8.7 and 9.6) and than in two samples of cancer patients in remission (9.4 and 10.0). Scores for the SSc sample were significantly lower (less fatigue) compared with two samples of cancer patients in palliative care (16.8 and 17.0). SSc GFI scores were similar to scores from patients with RA (13.4), AS (13.0) and SLE (13.1) and to scores from six studies of cancer patients in active treatment (11.1-13.5). CONCLUSIONS: The high levels of fatigue reported in SSc were similar to patients with varying types and treatment stages of cancer and patients with other rheumatic diseases when assessed with the GFI, demonstrating that fatigue warrants greater attention in SSc.

The relationship of dyspnoea to function and quality of life in systemic sclerosis.

AIM: Up to 50% of patients with systemic sclerosis (SSc) have complaints of dyspnoea. We evaluated the independent contributions of dyspnoea to function and health related quality of life (HRQoL) in SSc and also assessed the contributions of pulmonary hypertension, measured by the pulmonary artery systolic pressure (PASP), and interstitial lung disease, measured by the forced vital capacity (FVC), to dyspnoea. METHODS: We assessed dyspnoea, PASP, FVC, function and HRQoL in a cohort of unselected patients with SSc. Multiple linear regression was used to assess the independent contributions of dyspnoea, PASP and FVC to function and HRQoL, after controlling for possible confounding variables. RESULTS: A total of 194 patients with mean disease duration of 11.6 years were studied. Dyspnoea was a significant independent predictor of function and HRQoL. A model including age, gender, disease duration, disease severity and dyspnoea explained 33.3%, 10.6%, 39.2% and 29.4% of the variance of the Stanford Health Assessment Questionnaire, the Short-Form 36 (SF-36) mental component summary score, the SF-36 physical component summary score and the World Health Organization Disability Assessment Schedule II. PASP and FVC were significant independent predictors of dyspnoea but only 21.9% of the variance in dyspnoea was explained by age, gender, disease duration, FVC and PASP. The FVC was a significant independent predictor of function and HRQoL. CONCLUSION: In an unselected population of SSc patients, dyspnoea is a very important contributor to function and HRQoL. Interstitial lung disease, as measured by the FVC, contributes significantly to the sense of dyspnoea, function and HRQoL in SSc. Pulmonary hypertension, assessed echocardiographically by the PASP, predicts the degree of dyspnoea but not function and HRQoL in SSc.