An interim report of the Scleroderma Clinical Trials Consortium working groups

Baron, Murray; Kahaleh, Bashar; Bernstein, Elana J.; Chung, Lorinda; Clements, Philip J.; Denton, Christopher P.; Domsic, Robyn T.; Ferdowsi, Nava; Foeldvari, Ivan; Frech, Tracy; Gordon, Jessica; Hudson, Marie; Khanna, Dinesh; McMahan, Zsuzsannah; Merkel, Peter A.; Narain, Sonali; Nikpour, Mandana; Pauling, John D; Ross, Laura; Vergara, Antonia Maria Valenzuela; Vacca, Alessandra

doi:10.1177/2397198318783926

Cited by 14 publications

(10 citation statements)

References 64 publications

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“…Similar initiatives supported by the SCTC to create a working definition and classification criteria for primary SSc heart involvement are ongoing. 39 Future studies will evaluate for complementarity of these two definitions and/or any added value of combined use of both definitions in improving the accuracy and uniformity of SSc-pHI identification in clinical research.…”

Section: Discussionmentioning

confidence: 99%

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Buch¹

2021

Journal of Scleroderma and Related Disorders

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Introduction: Primary heart involvement in systemic sclerosis may cause morpho-functional and electrical cardiac abnormalities and is a common cause of death. The absence of a clear definition of primary heart involvement in systemic sclerosis limits our understanding and ability to focus on clinical research. We aimed to create an expert consensus definition for primary heart involvement in systemic sclerosis. Methods: A systematic literature review of cardiac involvement and manifestations in systemic sclerosis was conducted to inform an international and multi-disciplinary task force. In addition, the nominal group technique was used to derive a definition that was then subject to voting. A total of 16 clinical cases were evaluated to test face validity, feasibility, reliability and criterion validity of the newly created definition. Results: In total, 171 publications met eligibility criteria. Using the nominal group technique, experts added their opinion, provided statements to consider and ranked them to create the consensus definition, which received 100% agreement on face validity. A median 60(5–300) seconds was taken for the feasibility on a single case. Inter-rater agreement was moderate (mKappa (95% CI) = 0.56 (0.46–1.00) for the first round and 0.55 (0.44–1.00) for the second round) and intra-rater agreement was good (mKappa (95% CI) = 0.77 (0.47–1.00)). Criterion validity showed a 78 (73–84)% correctness versus gold standard. Conclusion: A preliminary primary heart involvement in systemic sclerosis consensus-based definition was created and partially validated, for use in future clinical research.

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Section: Discussionmentioning

confidence: 99%

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Buch¹

2021

Journal of Scleroderma and Related Disorders

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“…Composite scores like the revised European Scleroderma Research Group Activity Index have been proposed, but have not been widely accepted in the evaluation of disease activity (28). Novel approaches for assessing disease activity in SSc are currently under development (32).…”

Section: Why Is Disease-modifying Therapy a Challenge In Ssc?mentioning

confidence: 99%

“…'damage' is a challenge which currently is an area of investigation (32). Activity is defined as that component of disease severity that is largely reversible and may result in little or no damage in the future.…”

Section: How Should We Define Remission and Low Disease Activity (Lda) In Ssc?mentioning

confidence: 99%

Current and Future Outlook on Disease Modification and Defining Low Disease Activity in Systemic Sclerosis

Nagaraja

Matucci‐Cerinic

Fürst

et al. 2020

Arthritis & Rheumatology

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Systemic sclerosis (SSc) is an autoimmune rheumatic disease with heterogeneous clinical manifestations and a variable course in which the severity of the pathology dictates the disease prognosis and course. Among autoimmune rheumatic diseases, SSc has the highest mortality rate among all rheumatic diseases, though there are exciting new therapeutic targets that appear to halt the progression of SSc manifestations such as skin or lung fibrosis. In selected patients, high‐intensity regimens with autologous stem cell transplantation can favorably modify the course. In what was once thought to be an untreatable disease, targeted therapies have now changed the outlook of SSc to a treatable disorder. Herein, we discuss the targeted therapies modifying the outlook on selected organ involvement and creating opportunities for future treatment. We also present a framework for defining low disease activity in SSc.

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“…While not formally validated for primary RP, it has been successfully used in studies of mixed populations of patients 47. Recent work has highlighted limitations to the RCS diary and work is underway to develop novel patient-reported outcome instruments that might complement diary-based approaches in future clinical trials assessing the outcome of behaviour intervention on the severity and impact of RP 48–50…”

Section: Discussionmentioning

confidence: 99%

Behaviour change interventions for the management of Raynaud’s phenomenon: a systematic literature review

2018

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ObjectivesRaynaud’s phenomenon (RP) is a significant cause of morbidity. Vasodilator medications cause unwanted adverse effects, with behavioural and lifestyle changes forming the mainstay of self-management; this is difficult to implement successfully. The objectives of this study were to evaluate the efficacy of behaviour change interventions for RP and identify learning points for future treatment development.DesignSystematic literature review and narrative synthesis of findings.Data sourcesEMBASE, MEDLINE, Cochrane and PsycINFO were searched for eligible studies on 22 August 2017.Eligibility criteriaRandomised controlled trials (RCTs) of behaviour change interventions with at least one control comparator arm.Data extraction and synthesisStudy selection, data extraction and risk of bias were assessed independently by two reviewers, reaching consensus with a third when necessary. Primary outcomes of interest included severity/impact, frequency and duration of RP episodes, pain, disability, adverse events and study withdrawal.ResultsOf 638 articles retrieved, eight studies fulfilled criteria for inclusion. Biofeedback was the active behaviour change treatment arm for seven studies, with one study reporting a behavioural intervention. Studies were published 1978–2002; six were USA-based studies, one German and one Swedish. Using Cochrane Risk of Bias assessment, studies were assessed to be overall at high risk of bias, with the exception of one large RCT. The total sample included 495 participants (study median=29), with a median age of 39.5 years and preponderance towards females (73%). Five studies reported significant effects in primary outcomes of interest; however, due to missing data, relative efficacy of interventions could not be reliably assessed.ConclusionsThere is no evidence to support or refute claims of the efficacy of behaviour change interventions for the management of RP. There remains a strong case for developing and testing behaviour change interventions that focus on self-management; however, theoretical development and advancement in trial quality is imperative to underpin future work.PROSPERO registration numberCRD42017049643.

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An interim report of the Scleroderma Clinical Trials Consortium working groups

Cited by 14 publications

References 64 publications

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Primary systemic sclerosis heart involvement: A systematic literature review and preliminary data-driven, consensus-based WSF/HFA definition

Current and Future Outlook on Disease Modification and Defining Low Disease Activity in Systemic Sclerosis

Behaviour change interventions for the management of Raynaud’s phenomenon: a systematic literature review

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