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Introduction Systemic 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 analysis Eligible 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 dissemination We 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 number CRD42020219914.

Objectives Randomised 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.Methods The 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.Results 27 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.Conclusion Uptake 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.

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.

Objectives The Social Appearance Anxiety Scale (SAAS) is a 16-item questionnaire developed to evaluate fear of appearance-based evaluation by others. The primary objective of this research was to investigate the existence of differential item functioning (DIF) for the 16 SAAS items, comparing patients who completed the SAAS in English and French, either to confirm that scores are comparable or provide guidance on calculating comparable scores. A secondary research objective was to investigate the existence of DIF based on sex and disease status. A tertiary research objective was to assess DIF related to language, sex, and disease status on the recently developed SAAS-5.Design This was a cross-sectional analysis using baseline data from patients enrolled in the Scleroderma Patient-centred Intervention Network (SPIN).Setting SPIN patients included in the present study were enrolled at 43 centres in Canada, USA, UK, France and Australia, with questionnaires completed in April 2014 to July 2019.Participants 1640 SPIN patients completed the SAAS in French (n=600) or English (n=1040).Primary and secondary measures The SAAS was collected along with demographic and disease characteristics.Results Six items were identified with statistically significant language-based DIF, four with sex-based DIF and one with disease type-based DIF. However, factor scores before and after accounting for DIF were similar (Pearson correlation >0.99), and individual score differences were small. This was true for both the full and shortened versions of the SAAS.Conclusion SAAS and SAAS-5 scores are comparable across language, sex, and disease-type, despite small differences in how patients respond to some items.

Objectives The 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.Design Non-randomised feasibility trial.Setting North American patient organisations.Participants Current support group leaders or potential new leaders referred by patient organisations.Intervention The 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.Results All 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).Conclusion The 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 number NCT03508661

Objectives The Scleroderma Patient-centered Intervention Network (SPIN) Cohort uses the cohort multiple randomised controlled trial design to embed trials of online self-care interventions for people living with systemic sclerosis (SSc; scleroderma). To offer interventions to patients interested in using them, participants complete signalling items that query about the likelihood that patients would agree to participate in nine different hypothetical online programmes addressing common SSc-related problems. It is not known what factors influence patient-reported interest in participating in a particular online intervention and if intervention-specific signalling questions provide unique information or replicate broader characteristics, such as overall willingness to participate or self-efficacy. This study assessed factors that explain responses to intervention-specific signalling items.Design Cross-sectional survey.Setting SPIN Cohort participants enrolled at 42 centres from Canada, the USA, the UK, France, Spain and Mexico who completed study questionnaires from March 2014 to January 2018 were included.Measures Demographic and disease characteristics, self-efficacy and symptoms related to each specific intervention were completed in addition to signalling items. General likelihood of using interventions was calculating by taking the mean score of the remaining signalling questions.Participants 1060 participants with complete baseline data were included in the analyses.Results For all individual signalling questions, controlling for other variables, the mean of the remaining signalling questions was the strongest predictor (standardised regression coefficient β from 0.61 (sleep) to 0.80 (self-management)). Smaller, but statistically significant, associations were found with the symptom associated with the respective signalling question and with general self-efficacy for 7 of 9 signalling questions.Conclusions The main factor associated with patients’ interest in participating in a disease-specific online self-care intervention is their general interest in participating in online interventions. Factors that may influence this general interest should be explored and taken into consideration when inviting patients to try online interventions.