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Contains fulltext : 231157.pdf (Publisher’s version ) (Closed access) Objective: To support physical activity among people with systemic sclerosis (SSc; scleroderma), we sought to determine the (1) prevalence and importance of barriers and (2) likelihood of using possible facilitators. Methods: We invited 1,707 participants from an international SSc cohort to rate the (1) importance of 20 barriers (14 medical; 4 social or personal; 1 lifestyle; 1 environmental); and (2) likelihood of using 91 corresponding barrier-specific and 12 general facilitators. Results: Among 721 respondents, 13 barriers were experienced by ≥25% of participants, including 2 (fatigue, Raynaud's) rated 'important' or 'very important' by ≥50% of participants, 7 (joint stiffness and contractures, shortness of breath, gastrointestinal problems, difficulty grasping, pain, muscle weakness and mobility limitations, low motivation) by 26-50%, and 4 by 50% as 'likely' or 'very likely'. Conclusion: Medical-related physical activity barriers were common and considered important. Facilitators considered as most likely to be used involved adapting exercise, taking care of one’s body, keeping warm, and protecting skin. 11 p.

Background Systemic sclerosis (scleroderma; SSc) is a rare autoimmune connective tissue disease. We completed an initial feasibility trial of an online self-administered version of the Scleroderma Patient-centered Intervention Network Self-Management (SPIN-SELF) Program using the cohort multiple randomized controlled trial (RCT) design. Due to low intervention offer uptake, we will conduct a new feasibility trial with progression to full-scale trial, using a two-arm parallel, partially nested RCT design. The SPIN-SELF Program has also been revised to include facilitator-led videoconference group sessions in addition to online material. We will test the group-based intervention delivery format, then evaluate the effect of the SPIN-SELF Program on disease management self-efficacy (primary) and patient activation, social appearance anxiety, and functional health outcomes (secondary). Methods This study is a feasibility trial with progression to full-scale RCT, pending meeting pre-defined criteria, of the SPIN-SELF Program. Participants will be recruited from the ongoing SPIN Cohort (http://www.spinsclero.com/en/cohort) and via social media and partner patient organizations. Eligible participants must have SSc and low to moderate disease management self-efficacy (Self-Efficacy for Managing Chronic Disease (SEMCD) Scale score ≤ 7.0). Participants will be randomized (1:1 allocation) to the group-based SPIN-SELF Program or usual care for 3 months. The primary outcome in the full-scale trial will be disease management self-efficacy based on SEMCD Scale scores at 3 months post-randomization. Secondary outcomes include SEMCD scores 6 months post-randomization plus patient activation, social appearance anxiety, and functional health outcomes at 3 and 6 months post-randomization. We will include 40 participants to assess feasibility. At the end of the feasibility portion, stoppage criteria will be used to determine if the trial procedures or SPIN-SELF Program need important modifications, thereby requiring a re-set for the full-scale trial. Otherwise, the full-scale RCT will proceed, and outcome data from the feasibility portion will be utilized in the full-scale trial. In the full-scale RCT, 524 participants will be recruited. Discussion The SPIN-SELF Program may improve disease management self-efficacy, patient activation, social appearance anxiety, and functional health outcomes in people with SSc. SPIN works with partner patient organizations around the world to disseminate its programs free-of-charge. Trial registration ClinicalTrials.govNCT04246528. Registered on 27 January 2020

ObjectivesRandomised controlled trials conducted using cohorts and routinely collected data, including registries, electronic health records and administrative databases, are increasingly used in healthcare intervention research. A Consolidated Standards of Reporting Trials (CONSORT) statement extension for trials conducted using cohorts and routinely collected data (CONSORT-ROUTINE) has been developed with the goal of improving reporting quality. This article describes the processes and methods used to develop the extension and decisions made to arrive at the final checklist.MethodsThe development process involved five stages: (1) identification of the need for a reporting guideline and project launch; (2) conduct of a scoping review to identify possible modifications to CONSORT 2010 checklist items and possible new extension items; (3) a three-round modified Delphi study involving key stakeholders to gather feedback on the checklist; (4) a consensus meeting to finalise items to be included in the extension, followed by stakeholder piloting of the checklist; and (5) publication, dissemination and implementation of the final checklist.Results27 items were initially developed and rated in Delphi round 1, 13 items were rated in round 2 and 11 items were rated in round 3. Response rates for the Delphi study were 92 of 125 (74%) invited participants in round 1, 77 of 92 (84%) round 1 completers in round 2 and 62 of 77 (81%) round 2 completers in round 3. Twenty-seven members of the project team representing a variety of stakeholder groups attended the in-person consensus meeting. The final checklist includes five new items and eight modified items. The extension Explanation & Elaboration document further clarifies aspects that are important to report.ConclusionUptake of CONSORT-ROUTINE and accompanying Explanation & Elaboration document will improve conduct of trials, as well as the transparency and completeness of reporting of trials conducted using cohorts and routinely collected data.

IntroductionSystemic sclerosis (SSc; scleroderma) is a rare, chronic, autoimmune disease with a high level of burden, a significant impact on the ability to carry out daily activities, and a considerable negative impact on health-related quality of life. Non-pharmacological interventions could be provided to potentially improve mental and physical health outcomes. However, the effectiveness of non-pharmacological interventions on health and well-being among individuals with SSc has not been well established. The proposed living systematic review aims to identify and evaluate randomised controlled trial (RCT) evidence on the effectiveness of non-pharmacological and non-surgical interventions on mental and physical health outcomes and on the delivery of such services in SSc.Methods and analysisEligible studies will be RCTs that examine non-pharmacological and non-surgical interventions aimed at improving health outcomes among individuals with SSc or the delivery of services intended to improve healthcare or support of people with SSc (eg, support groups). All RCTs included in a previous systematic review that sought studies published between 1990 and March 2014 will be evaluated for inclusion. Additional trials will be sought from January 2014 onwards using a similar, augmented search strategy developed by a health sciences librarian. We will search the MEDLINE, Embase, CINAHL, PsycINFO, Cochrane Library and Web of Science databases and will not restrict by language. Two independent reviewers will determine the eligibility of identified RCTs and will extract data using a prespecified standardised form in DistillerSR. Meta-analyses will be considered if ≥2 eligible RCTs report similar non-pharmacological interventions and comparable health outcomes. We will conduct a qualitative synthesis for interventions that cannot be synthesised via meta-analysis.Ethics and disseminationWe will post initial and ongoing results via a website, publish results periodically via peer-reviewed journal publication, and present results at patient-oriented events.PROSPERO registration numberCRD42020219914.

Randomised controlled trials are increasingly conducted as embedded, nested, or using cohorts or routinely collected data, including registries, electronic health records, and administrative databases, to assess if participants are eligible for the trial and to facilitate recruitment, to deliver an embedded intervention, to collect trial outcome data, or a combination of these purposes. This report presents the Consolidated Standards of Reporting Trials (CONSORT) extension for randomised controlled trials conducted using cohorts and routinely collected data (CONSORT-ROUTINE). The extension was developed to look at the unique characteristics of trials conducted with these types of data with the goal of improving reporting quality in the long term by setting standards early in the process of uptake of these trial designs. The extension was developed with a sequential approach, including a Delphi survey, a consensus meeting, and piloting of the checklist. The checklist was informed by the CONSORT 2010 statement and two reporting guidelines for observational studies, the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement and the REporting of studies Conducted using Observational Routinely collected Data (RECORD) statement. The extension includes eight items modified from the CONSORT 2010 statement and five new items. Reporting items with explanations and examples are provided, including key aspects of trials conducted using cohorts or routinely collected data that require specific reporting considerations.

Summary Background Pain is an important and detrimental feature of systemic sclerosis but is often overlooked or deprioritised in research and clinical care. Raynaud's phenomenon, arthritis, and cutaneous ulcers are among the commonly reported disease manifestations of systemic sclerosis that could be associated with pain. We aimed to assess levels of pain intensity and interference and to evaluate disease factors associated with pain intensity and interference. Methods In this multicentre cross-sectional study, participants from the Scleroderma Patient-centered Intervention Network cohort who completed pain intensity and interference measures (Patient Reported Outcomes Information System-29 profile, version 2·0) as part of baseline assessments were included. Patients were recruited from 46 centres in Australia, Canada, France, Mexico, Spain, the UK, and the USA between April 15, 2014, and Jan 7, 2020. Eligible patients included those aged 18 years or older who met the criteria for systemic sclerosis devised by the American College of Rheumatology and the European League Against Rheumatism. Associations of pain intensity and pain interference with systemic sclerosis-related variables and overlap syndromes, controlling for sociodemographic variables, were assessed with multiple linear regression. Continuous independent variables were standardised. Findings Among 2157 participants with systemic sclerosis (268 [12%] males and 1889 [88%] females), 1870 (87%) reported mild, moderate, or severe pain (defined as ≥1 on a 0 to 10 scale), and 815 (38%) reported moderate or severe pain (defined as ≥5). Moreover, 757 (35%) participants reported moderate or severe pain interference. Greater pain intensity was independently associated with female sex (0·58 points [95% CI 0·26–0·90]), non-White race or ethnicity (0·50 points [0·21–0·79]), fewer years in formal education (0·30 points per SD [0·19–0·41]), country (reference: USA; Canada: 0·29 points [0·01–0·57] and UK: 0·58 points [0·21–0·95]), greater body-mass index (0·35 points per SD [0·24–0·45]); joint contractures (0·67 points [0·39–0·94]), digital ulcers (0·33 points [0·10–0·55]), gastrointestinal involvement (0·66 points [0·33–0·98]), skin involvement (measured using modified Rodnan skin score; 0·22 points per SD [0·10–0·35]), rheumatoid arthritis (0·96 points [0·50–1·43]), and Sjogren's syndrome (0·42 points [0·01–0·83]). Pain interference results were similar. Interpretation Pain is common among people with systemic sclerosis. Controlling for sociodemographic variables, greater pain was associated with multiple systemic sclerosis-related manifestations, including joint contractures, digital ulcers, gastrointestinal involvement, skin involvement, and the presence of overlap syndromes. Health-care providers should work with patients to address pain, including identifying and addressing systemic sclerosis manifestations associated with their pain, and supporting behavioural approaches to minimise impact on function and quality of life. Funding Canadian Institutes of Health Research, Arthritis Society, The Lady Davis Institute for Medical Research of the Jewish General Hospital, Jewish General Hospital Foundation, McGill University, Scleroderma Society of Ontario, Scleroderma Canada, Sclerodermie Quebec, Scleroderma Manitoba, Scleroderma Atlantic, Scleroderma Association of BC, Scleroderma SASK, Scleroderma Australia, Scleroderma New South Wales, Scleroderma Victoria, and Scleroderma Queensland.

Background Systemic sclerosis (SSc), or scleroderma, is a rare disease that often results in significant disruptions to activities of daily living and can negatively affect physical and psychological well-being. Because there is no known cure, SSc treatment focuses on reducing symptoms and disability and improving health-related quality of life (HRQoL). Self-management programs are known to increase self-efficacy for disease management in many chronic diseases. The Scleroderma Patient-centered Intervention Network (SPIN) developed a Web-based self-management program (SPIN self-management; SPIN-SELF) to increase self-efficacy for disease management and to improve HRQoL for patients with SSc. Objective The proposed study aims to assess the feasibility of conducting a full-scale randomized controlled trial (RCT) of the SPIN-SELF program by evaluating the trial implementation processes, required resources and management, scientific aspects, and participant acceptability and usage of the SPIN-SELF program. Methods The SPIN-SELF feasibility trial will be conducted via the SPIN Cohort. The SPIN Cohort was developed as a framework for embedded pragmatic trials using the cohort multiple RCT design. In total, 40 English-speaking SPIN Cohort participants with low disease management self-efficacy (Self-Efficacy for Managing Chronic Disease Scale score ≤7), who have indicated interest in using a Web-based self-management program, will be randomized with a 3:2 ratio into the SPIN-SELF program or usual care for 3 months. Feasibility outcomes include trial implementation processes, required resources and management, scientific aspects, and patient acceptability and usage of the SPIN-SELF program. Results Enrollment of the 40 participants occurred between July 5, 2019, and July 27, 2019. By November 25, 2019, data collection of trial outcomes was completed. Data analysis is underway, and results are expected to be published in 2020. Conclusions The SPIN-SELF program is a self-help tool that may improve disease-management self-efficacy and improve HRQoL in patients with SSc. The SPIN-SELF feasibility trial will ensure that trial methodology is robust, feasible, and consistent with trial participant expectations. The results will guide adjustments that need to be implemented before undertaking a full-scale RCT of the SPIN-SELF program. International Registered Report Identifier (IRRID) DERR1-10.2196/16799

Contains fulltext : 175251.pdf (Publisher’s version ) (Closed access) Objective. The Patient-Reported Outcomes Measurement Information System (PROMIS)-29 assesses seven health-related quality of life domains plus pain intensity. The objective was to examine PROMIS-29v2 validity and explore clinical associations in patients with SSc. Methods. English-speaking SSc patients in the Scleroderma Patient-centered Intervention Network Cohort from 26 sites in Canada, the USA and the UK completed the PROMIS-29v2 between July 2014 and November 2015. Enrolling physicians provided medical data. To examine convergent validity, hypotheses on the direction and magnitude of correlations with legacy measures were tested. For clinical associations, t-tests were conducted for dichotomous variables and PROMIS-29v2 domain scores. Effect sizes (ESs) were labelled as small (0.75). Results. There were 696 patients (87% female), mean (s.d.) disease duration 11.6 (8.7) years, 57% with limited cutaneous subtype. Validity indices were consistent with seven of nine hypotheses (|r| =0.51-0.87, P < 0.001), with minor divergence for two hypotheses. Gastrointestinal involvement was associated with significantly worse outcomes for all eight PROMIS-29v2 domains (moderate or moderate to large ES in six of eight). Presence of joint contractures was associated with significant decrements in seven domains (small or small to moderate ESs). Skin thickening, diffuse cutaneous subtype and presence of overlap syndromes were significantly associated (small or small to moderate ESs) with five or six domains. Conclusion. This study further establishes the validity of the PROMIS-29v2 in SSc and underlines the importance of gastrointestinal symptoms and joint contractures in reduced health-related quality of life. 10 p.

Background:The SENSCIS® trial (2015–18) was a large clinical trial (n=576) investigating the efficacy and safety of nintedanib in patients with systemic sclerosis-associated interstitial lung disease.1 The clinical research sponsors (CRS) collaborated with the scleroderma patient community advisory board (CAB) regarding the design, implementation and conduct of the trial.2 As part of this collaboration, the CRS and CAB developed a post-trial survey for SENSCIS® participants. The use of the developed patient-centric materials was optional for the sites.Objectives:The objectives of the SENSCIS® post-trial survey were to gain experience in collecting real-world information and trial satisfaction data from patients to inform and improve future patient centric clinical research.Methods:SENSCIS® trial participants who were involved in the extension trial SENSCIS®-ON completed a post-trial survey covering nine multiple-choice questions about three main topics:[1]Recruitment – Where do patients usually search for clinical trials and how did they become aware of SENSCIS®?[2]Motivation & Retention – What motivated patients to start and continue participation in SENSCIS®?[3]Challenges & Wishes – What were the challenges during trial participation and how can future clinical trials be improved regarding patient centricity?Results:A total of 125 participants completed all survey questions. Participants could select more than one option. A total of 51 patients reported that they are usually not actively looking for trials. For those actively searching, the most common sources to learn about trials were specialists/general practitioners (GPs) (46 patients) and internet search engines (20 patients), followed by patient organisations (12 patients). Of note, 78 patients would pay attention to printed materials, such as a card/flyer/poster in a doctor’s office and get in touch with a trial/study site.Back in 2015–2017, during recruitment for the SENSCIS® trial, the majority of the patients who answered the survey were made aware via their specialist/GP (116 patients), whereas 5 were made aware via patient organisations and 4 via the internet.The most frequent motivations to join the trial were ‘hope to receive an improved therapy’ (98 patients), to help other patients (64 patients), and on the recommendation of their specialist/GP (81 patients). Similarly, the most liked aspects of the trial were the ‘opportunity to receive an improved therapy’ (92 patients) and ‘to support the development of an improved therapy for my illness’ (90 patients). More than half of patients reported ‘continuous observation of general health’ (72 patients) and ‘advice from GPs/specialists’ (71 patients) as motivation to stay in the trial (Figure 1).‘Concerns about side effects’ (72 patients) and ‘not knowing whether the trial medication will work for me’ (63 patients) were reported as the least liked aspects of the trial. Travel to the site was reported as a challenge by 21 patients.To improve clinical trials, patients requested more patient-friendly information (50 patients) and multiple formats of information material (46 patients). Finally, 48 patients expressed the desire to communicate with other trial participants.Conclusion:The SENSCIS® post-trial survey is a unique approach to receive real-world feedback from trial participants, and these pilot data will help improve future clinical trials and communication. The results highlight the importance of reaching patients who may not be actively looking for clinical trials.Figure 1.Motivation to stay in the SENSCIS® trial1,21More than one option could be selected.2Data collected on 9th January 2021References:[1]Distler O et al. N Engl J Med. 2019 Jun 27;380(26):2518-2528. doi: 10.1056/NEJMoa1903076.[2]Roennow A et al. BMJ Open. 2020 Dec 16;10(12):e039473. doi: 10.1136/bmjopen-2020-039473.Acknowledgements:Sue Farrington (Federation of European Scleroderma Associations [FESCA] Belgium), Luke Evnin (Scleroderma Research Foundation, United States), Beatriz Garcia (Asociacion Espanola de Esclerodermia, Spain), Catarina Leite (Associacao Portuguesa de Doentes com Esclerodermia, Portugal), Alison Zheng (Chinese Organisation for Scleroderma), Matea Perković Popović (Hrvatska udruga oboljelih od sklerodermije, Croatia), Tina Ampudia (Asociacion Mexicana de Orientacion Apoyo y Lucha Contra la Esclerodermia, AC, Mexico), Stephanie Munoz (Norsk Revmatikerforbund, Diagnosegruppen for Systemisk Sklerose, Norway), Monica Holmner (Reumatikerförbundet Riksföreningen för systemisk skleros, Sweden).Disclosure of Interests:Ilaria Galetti: None declared, EDITH BROWN: None declared, Ann Kennedy Consultant of: I have been a member of the CAB (Community Patient Advisory Board) described in the accompanying abstract under discussion. My patient organisation has been paid for its participation in the CAB., Grant/research support from: It is not myself personally, but FESCA (Federation of European Scleroderma Associations) aisbl., that has received project grants for awareness raising and education. I was President of this Federation., Robert J Riggs: None declared, Annelise Roennow: None declared, Maureen Sauvé: None declared, Joep Welling Speakers bureau: BI MIDI and BI International, Sanofi, Henrik Finnern Employee of: I am employee of Boehringer Ingelheim International GmbH, Annie Gilbert Consultant of: I am a paid consultant for Bohringer Ingelheim since 2016, Martina Gahlemann Employee of: I am employed by Boehringer Ingelheim (Schweiz) GmbH, Basel, Switzerland, Wiebke Sauter Employee of: I am employer of Boehringer-Ingelheim

Introduction Transparent collaborations between patient organisations (POs) and clinical research sponsors (CRS) can identify and address the unmet needs of patients and caregivers. These insights can improve clinical trial participant experience and delivery of medical innovations necessary to advance health outcomes and standards of care. We share our experiences from such a collaboration undertaken surrounding the SENSCIS® clinical trial (NCT02597933), and discuss its impact during, and legacy beyond, the trial.Summary We describe the establishment of a community advisory board (CAB): a transparent, multiyear collaboration between the scleroderma patient community and a CRS. We present shared learnings from the collaboration, which is split into three main areas: (1) the implementation and conduct of the clinical trial; (2) analysis and dissemination of the results; and (3) aspects of the collaboration not related to the trial.1. The scleroderma CAB reviewed and provided advice on trial conduct and reporting. This led to the improvement and optimisation of trial procedures; meaningful, patient-focused adaptations were made to address challenges relevant to scleroderma-associated interstitial lung disease patients.2. To ensure that results of the trial were accessible to lay audiences and patients, written lay summaries were developed by the trial sponsor with valuable input from the CAB to ensure that language and figures were understandable.3. The CAB and the CRS also collaborated to co-develop opening tools for medication blister packs and bottles. In addition, to raise disease awareness among physicians, patients and caregivers, educational materials to improve diagnosis and management of scleroderma were co-created and delivered by the CAB and CRS.Conclusions This collaboration between POs and a CRS, in a rare disease condition, led to meaningful improvements in patient safety, comfort and self-management and addressed information needs. This collaboration may serve as a template of best practice for future collaborations between POs, research sponsors and other healthcare stakeholders.

Objective: To develop and validate a short form of the Cochin Hand Function Scale (CHFS), which measures hand disability, for use in systemic sclerosis, using objective criteria and reproducible techniques. Methods: Responses on the 18-item CHFS were obtained from English-speaking patients enrolled in the Scleroderma Patient-Centered Intervention Network Cohort. CHFS unidimensionality was verified using confirmatory factor analysis, and an item response theory model was fit to CHFS items. Optimal test assembly (OTA) methods identified a maximally precise short form for each possible form length between 1 and 17 items. The final short form selected was the form with the least number of items that maintained statistically equivalent convergent validity, compared to the full-length CHFS, with the Health Assessment Questionnaire (HAQ) disability index (DI) and the physical function domain of the 29-item Patient-Reported Outcomes Measurement Information System (PROMIS-29). Results: There were 601 patients included. A 6-item short form of the CHFS (CHFS-6) was selected. The CHFS-6 had a Cronbach's alpha of 0.93. Correlations of the CHFS-6 summed score with HAQ DI (r = 0.79) and PROMIS-29 physical function (r = -0.54) were statistically equivalent to the CHFS (r = 0.81 and r = -0.56). The correlation with the full CHFS was high (r = 0.98). Conclusion: The OTA procedure generated a valid short form of the CHFS with minimal loss of information compared to the full-length form. The OTA method used was based on objective, prespecified criteria, but should be further studied for viability as a general procedure for shortening patient-reported outcome measures in health research.

Objective: Self-management programs for patients with chronic illnesses, including rheumatic diseases, seek to enhance self-efficacy for performing health management behaviors. No measure of self-efficacy has been validated for patients with systemic sclerosis (SSc; scleroderma). The objective of this study was to assess the validity and internal consistency reliability of the Self-Efficacy for Managing Chronic Disease (SEMCD) scale in SSc. Methods: English-speaking SSc patients enrolled in the Scleroderma Patient-centered Intervention Network Cohort who completed the SEMCD scale at their baseline assessment between March 2014 and June 2015 were included. Patients were enrolled from 21 sites in Canada, the US, and the UK. Confirmatory factor analysis (CFA) was used to evaluate the factor structure of the SEMCD scale. Cronbach's alpha was calculated to assess internal consistency reliability. Hypotheses on the direction and magnitude of Pearson's correlations with psychological and physical outcome measures were formulated and tested to examine convergent validity. Results: A total of 553 patients were included. CFA supported the single-factor structure of the SEMCD scale (Tucker Lewis Index = 0.99, comparative fit index = 0.99, root mean square error of approximation = 0.10). Internal consistency was high (alpha = 0.93), and correlations with measures of psychological and physical functioning were moderate to large (|r| = 0.48-0.67, P

IntroductionRandomised controlled trials (RCTs) conducted using cohorts and routinely collected health data, including registries, electronic health records and administrative databases, are increasingly used in healthcare intervention research. The development of an extension of the CONsolidated Standards of Reporting Trials (CONSORT) statement for RCTs using cohorts and routinely collected health data is being undertaken with the goal of improving reporting quality by setting standards early in the process of uptake of these designs. To develop this extension to the CONSORT statement, a scoping review will be conducted to identify potential modifications or clarifications of existing reporting guideline items, as well as additional items needed for reporting RCTs using cohorts and routinely collected health data.Methods and analysisIn separate searches, we will seek publications on methods or reporting or that describe protocols or results from RCTs using cohorts, registries, electronic health records and administrative databases. Data sources will include Medline and the Cochrane Methodology Register. For each of the four main types of RCTs using cohorts and routinely collected health data, separately, two investigators will independently review included publications to extract potential checklist items. A potential item will either modify an existing CONSORT 2010, Strengthening the Reporting of Observational Studies in Epidemiology or REporting of studies Conducted using Observational Routinely collected health Data item or will be proposed as a new item. Additionally, we will identify examples of good reporting in RCTs using cohorts and routinely collected health data.Ethics and disseminationThe proposed scoping review will help guide the development of the CONSORT extension statement for RCTs conducted using cohorts and routinely collected health data.

Contains fulltext : 197751.pdf (Publisher’s version ) (Open Access) Background: Randomized controlled trials (RCTs) are often complex and expensive to perform. Less than one third achieve planned recruitment targets, follow-up can be labor-intensive, and many have limited real-world generalizability. Designs for RCTs conducted using cohorts and routinely collected health data, including registries, electronic health records, and administrative databases, have been proposed to address these challenges and are being rapidly adopted. These designs, however, are relatively recent innovations, and published RCT reports often do not describe important aspects of their methodology in a standardized way. Our objective is to extend the Consolidated Standards of Reporting Trials (CONSORT) statement with a consensus-driven reporting guideline for RCTs using cohorts and routinely collected health data. Methods: The development of this CONSORT extension will consist of five phases. Phase 1 (completed) consisted of the project launch, including fundraising, the establishment of a research team, and development of a conceptual framework. In phase 2, a systematic review will be performed to identify publications (1) that describe methods or reporting considerations for RCTs conducted using cohorts and routinely collected health data or (2) that are protocols or report results from such RCTs. An initial "long list" of possible modifications to CONSORT checklist items and possible new items for the reporting guideline will be generated based on the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) and The REporting of studies Conducted using Observational Routinely-collected health Data (RECORD) statements. Additional possible modifications and new items will be identified based on the results of the systematic review. Phase 3 will consist of a three-round Delphi exercise with methods and content experts to evaluate the "long list" and generate a "short list" of key items. In phase 4, these items will serve as the basis for an in-person consensus meeting to finalize a core set of items to be included in the reporting guideline and checklist. Phase 5 will involve drafting the checklist and elaboration-explanation documents, and dissemination and implementation of the guideline. Discussion: Development of this CONSORT extension will contribute to more transparent reporting of RCTs conducted using cohorts and routinely collected health data. 9 p.

Introduction: Individuals with visible differences due to medical conditions, such as systemic sclerosis (SSc; scleroderma), have reported difficulty navigating social situations because of issues such as staring, invasive questions, and rude comments. Fears or anxiety linked to situations in which a person interacts with others is known as social interaction anxiety. However, there exists no validated measurement tool to examine social interaction anxiety in rheumatologic conditions. Methods: The present study examines the reliability (internal consistency) and validity (structural and convergent) of the Social Interaction Anxiety Scale-6 (SIAS-6) in a sample of 802 individuals with SSc, and compares these psychometric properties across limited and diffuse subtypes of the disease. Multi-group confirmatory factor analysis was used to examine the factor structure of the SIAS-6 in patients with both limited and diffuse SSc. Results: A one-factor structure was found to fit well for individuals with SSc with both limited and diffuse disease. The measure demonstrated strong internal consistency reliability and convergent validity with relevant measures in expected magnitudes and directions. Conclusions: The SIAS-6 is a psychometrically robust measure that can confidently be used in SSc populations to examine social interaction anxiety. Moreover, scores can meaningfully be compared between patients with limited and diffuse disease., MD Anderson Cancer Center - University of Texas MD Anderson Cancer Center(undefined), Financial support: The Scleroderma Patient-centered Intervention Network (SPIN) is funded by a Canadian Institutes of Health Research (CIHR) Emerging Team Grant for Rare Diseases (PI, Thombs; TR3-119192). In addition to CIHR funding, SPIN has received institutional contributions from the Lady Davis Institute for Medical Research of the Jewish General Hospital, Montréal, Canada and from McGill University, Montréal, Canada. SPIN has also received support from the Scleroderma Society of Ontario, Scleroderma Canada, and Sclérodermie Québec. Ms. Gholizadeh’s work on this project was supported by a Rheumatology Research Foundation: Health Professional Research Preceptorship. Dr. Kwakkenbos was supported by a CIHR Banting Postdoctoral Fellowship. Ms. Jewett was supported by a CIHR Doctoral Research Award. Dr. Thombs was supported by an Investigator Salary Award from the Arthritis Society

ObjectivesThe Scleroderma Patient-centered Intervention Network—Scleroderma Support group Leader EDucation (SPIN-SSLED) Programme was designed to improve confidence and self-efficacy and to reduce burden for support group leaders. Objectives were to (1) evaluate feasibility of programme delivery, including required resources, management issues and scientific aspects (eg, performance of outcome measures) and (2) assess user satisfaction and identify any modifications needed to improve programme content or delivery based on participant feedback.DesignNon-randomised feasibility trial.SettingNorth American patient organisations.ParticipantsCurrent support group leaders or potential new leaders referred by patient organisations.InterventionThe programme included 13 modules delivered live via videoconference over 3 months (April to July 2018) in 60 to 90 min sessions.Outcome measures(1) Elements of feasibility, including enrolment and consent procedures, percentage of referred group leaders who consented to participate, session attendance and technical support requirements; (2) programme usability, understandability, organisation and clarity; (3) leader satisfaction with the programme and (4) planned trial outcome measures, including support group leader self-efficacy, burnout, emotional distress and physical function.ResultsAll 12 referred potential participants consented to enrol, and 10 were included in two training groups of five participants each. Participants attended 95% of sessions. Required technical support was minimal, and videoconferencing technology functioned well. Overall programme satisfaction rating was 9.4/10. Mean item rating on the eight items of the Client Satisfaction Questionnaire-8 was 3.83 (1=low satisfaction; 4=high satisfaction). Pre-post scores on the Scleroderma Support Group Leader Self-efficacy Scale increased by 1.7 SDs (large effect); scores on burnout, emotional distress and physical function improved by 0.44, 0.38 and 0.45 SDs (moderate effects).ConclusionThe SPIN-SSLED Programme was feasibly delivered, including management, resource and scientific aspects. Participant satisfaction was high. The programme is ready to be tested in a full-scale randomised controlled trial.Trial registration numberNCT03508661

Item does not contain fulltext Body concealment is an important component of appearance distress for individuals with disfiguring conditions, including scleroderma. The objective was to replicate the validation study of the Body Concealment Scale for Scleroderma (BCSS) among 897 scleroderma patients. The factor structure of the BCSS was evaluated using confirmatory factor analysis and the Multiple-Indicator Multiple-Cause model examined differential item functioning of SWAP items for sex and age. Internal consistency reliability was assessed via Cronbach's alpha. Construct validity was assessed by comparing the BCSS with a measure of body image distress and measures of mental health and pain intensity. Results replicated the original validation study, where a bifactor model provided the best fit. The BCSS demonstrated strong internal consistency reliability and construct validity. Findings further support the BCSS as a valid measure of body concealment in scleroderma and provide new evidence that scores can be compared and combined across sexes and ages. 8 p.

To develop and validate a short form of the Cochin Hand Function Scale (CHFS), which measures hand disability, for use in systemic sclerosis, using objective criteria and reproducible techniques.Responses on the 18-item CHFS were obtained from English-speaking patients enrolled in the Scleroderma Patient-Centered Intervention Network Cohort. CHFS unidimensionality was verified using confirmatory factor analysis, and an item response theory model was fit to CHFS items. Optimal test assembly (OTA) methods identified a maximally precise short form for each possible form length between 1 and 17 items. The final short form selected was the form with the least number of items that maintained statistically equivalent convergent validity, compared to the full-length CHFS, with the Health Assessment Questionnaire (HAQ) disability index (DI) and the physical function domain of the 29-item Patient-Reported Outcomes Measurement Information System (PROMIS-29).There were 601 patients included. A 6-item short form of the CHFS (CHFS-6) was selected. The CHFS-6 had a Cronbach's alpha of 0.93. Correlations of the CHFS-6 summed score with HAQ DI (r = 0.79) and PROMIS-29 physical function (r = -0.54) were statistically equivalent to the CHFS (r = 0.81 and r = -0.56). The correlation with the full CHFS was high (r = 0.98).The OTA procedure generated a valid short form of the CHFS with minimal loss of information compared to the full-length form. The OTA method used was based on objective, prespecified criteria, but should be further studied for viability as a general procedure for shortening patient-reported outcome measures in health research.

Self-management programs for patients with chronic illnesses, including rheumatic diseases, seek to enhance self-efficacy for performing health management behaviors. No measure of self-efficacy has been validated for patients with systemic sclerosis (SSc; scleroderma). The objective of this study was to assess the validity and internal consistency reliability of the Self-Efficacy for Managing Chronic Disease (SEMCD) scale in SSc.English-speaking SSc patients enrolled in the Scleroderma Patient-centered Intervention Network Cohort who completed the SEMCD scale at their baseline assessment between March 2014 and June 2015 were included. Patients were enrolled from 21 sites in Canada, the US, and the UK. Confirmatory factor analysis (CFA) was used to evaluate the factor structure of the SEMCD scale. Cronbach's alpha was calculated to assess internal consistency reliability. Hypotheses on the direction and magnitude of Pearson's correlations with psychological and physical outcome measures were formulated and tested to examine convergent validity.A total of 553 patients were included. CFA supported the single-factor structure of the SEMCD scale (Tucker Lewis Index = 0.99, comparative fit index = 0.99, root mean square error of approximation = 0.10). Internal consistency was high (α = 0.93), and correlations with measures of psychological and physical functioning were moderate to large (|r| = 0.48-0.67, P 0.001), confirming study hypotheses.Scores from the SEMCD scale are valid for measuring self-efficacy in patients with SSc, and results support using the scale as an outcome measure to evaluate the effectiveness of self-management programs in SSc.

Introduction: Psychosocial and rehabilitation interventions are increasingly used to attenuate disability and improve health-related quality of life (HRQL) in chronic diseases, but are typically not available for patients with rare diseases. Conducting rigorous, adequately powered trials of these interventions for patients with rare diseases is difficult. The Scleroderma Patient-centered Intervention Network (SPIN) is an international collaboration of patient organisations, clinicians and researchers. The aim of SPIN is to develop a research infrastructure to test accessible, low-cost self-guided online interventions to reduce disability and improve HRQL for people living with the rare disease systemic sclerosis (SSc or scleroderma). Once tested, effective interventions will be made accessible through patient organisations partnering with SPIN. Methods and analysis: SPIN will employ the cohort multiple randomised controlled trial (cmRCT) design, in which patients consent to participate in a cohort for ongoing data collection. The aim is to recruit 1500– 2000 patients from centres across the world within a period of 5 years (2013–2018). Eligible participants are persons ≥18 years of age with a diagnosis of SSc. In addition to baseline medical data, participants will complete patient-reported outcome measures every 3 months. Upon enrolment in the cohort, patients will consent to be contacted in the future to participate in intervention research and to allow their data to be used for comparison purposes for interventions tested with other cohort participants. Once nterventions are developed, patients from the cohort will be randomly selected and offered interventions as part of pragmatic RCTs. Outcomes from patients offered interventions will be compared with outcomes from trial-eligible patients who are not offered the interventions. Ethics and dissemination: The use of the cmRCT design, the development of self-guided online interventions and partnerships with patient organisations will allow SPIN to develop, rigourously test and effectively disseminate psychosocial and rehabilitation interventions for people with SSc., (undefined)

Background The Patient-Reported Outcomes Measurement Information System (PROMIS®) initiative is a cooperative research program designed to develop, evaluate, and standardize item banks to measure patient-reported outcomes across medical conditions. The PROMIS-29 measure contains 29 items, which include four items each for domains reflecting physical function, anxiety, depression, fatigue, sleep disturbance, pain interference, and ability to perform social roles, plus a single item on pain intensity. Scores are standardized with a mean of 50 and standard deviation (SD) of 10. Higher scores represent more of the domain being measured (e.g., greater sleep disturbance, greater ability to perform social roles). Objectives To examine feasibility and construct validity of the PROMIS29 in patients with systemic sclerosis (SSc) enrolled in a large multinational study. Methods English-speaking patients with SSc and ≥18 years of age were enrolled in the Scleroderma Patient-centered Intervention Network (SPIN) Cohort between April 2014 and January 2015 from 17 centers across Canada, the USA and the UK. Baseline medical data are provided by the enrolling physician, and SPIN Cohort patients complete outcome measures online every 3 months. Patients were included in the analyses if they completed at least one PROMIS-29 domain at baseline. Floor and ceiling effects were defined as >15% of patients having the lowest or highest possible domain score, respectively. To examine convergent validity of domains, hypotheses were formulated a-priori about the associations of domains and legacy measures. The magnitude of the correlations was interpreted as small (|r| ≤0.3), moderate (0.3 Results In total, 376 patients were included in analyses. Mean age was 55.1 years (SD=11.9) and mean time since onset of the first non-Raynaud symptom was 14.4 years (SD=11.8). Most patients were female (n=329, 87.5%) and diagnosed with limited SSc (n=207, 55.1%). Means for the PROMIS-29 domains were: function 42.4 (SD=8.7), anxiety 51.8 (SD=10.1), depression 50.9 (SD=9.3), fatigue 56.2 (SD=11.4), sleep 52.0 (SD=5.2), roles 47.4 (SD=9.5), pain interference 55.9 (SD=9.8), and pain intensity 3.8 (SD=2.7). There was a floor effect for anxiety (33.8%) and depression (37.7%), and ceiling effects for function (19.1%), roles (15.1%) and pain interference (24%). Most hypotheses were confirmed (7 of 9) and all were in the hypothesized direction (Table 1). Conclusions Results of our study support the construct validity of the PROMIS-29 in patients with SSc. Future studies should examine the influence of floor- and ceiling effects for some domains, as well as other psychometric properties of the measure. Disclosure of Interest None declared