Background/Purpose: The OMERACT Scleroderma Vascular Disease Working Group sought to identify essential core outcome domains for inclusion in clinical trials focusing on Raynaud’s phenomenon (RP) and digital ulcers (DUs) caused by systemic sclerosis (SSc). This effort aimed to gather insights from patients and other stakeholders, ensuring their perspectives are reflected in the development of a Core Outcome Set.

Methods: Candidate items identified from previous qualitative work and systematic literature reviews were included in separate Delphi surveys for RP and DUs. The surveys were in English and conducted in parallel. Participants were invited by email: patients through international patient advocacy groups, and other stakeholders, including physicians and others with experience in treating patients with SSc-related RP and DUs and/or conducting clinical trials in these disorders. Patients completed the surveys for RP and DUs only if they had lived experience of RP and DUs, respectively. Core domains were defined as those reaching group consensus (≥70% ratings of ‘critical’ in both patient and other stakeholder groups) in the third/final Delphi exercise.

Results: The 3-round Delphi surveys were completed by 62 patients and 132 others from 39 countries for RP, and by 20 patients and 105 others from 36 countries for DUs.

For RP, group consensus for core domains was met for i) pathophysiological manifestations core area: severity and frequency of RP attacks; and ii) life impact core area: items grouped under the target domain of function, adaptation required to manage life with RP, and impact on health-related quality of life (HRQoL). Pain, duration of RP attacks, patient global assessment (PtGA), and need for hospitalization (resource usecore area) were considered critical by other stakeholders but not by patients. Conversely, patients but not other stakeholders considered coldness to be critical. Assessment of perfusion and temperature (biomarkers category) did not meet the threshold for inclusion by either group but assessment of microvasculature was raised for further consideration by the Working Group.

For DUs, group consensus for core domains was met for i) pathophysiological manifestations core area: pain, number, global burden, healing of DUs, and physician global assessment; and ii) life impact core area: items grouped under the target domain of function, adaptation required to manage life with DUs, and impact on HRQoL; and iii) resource use core area: items grouped under hospitalization or urgent intervention. Digital sensitivity, impact on emotional well-being, and assessment of microvasculature were considered critical by patients but not by other stakeholders. PtGA was considered critical by others but not patients.

Conclusion: Patients with SSc and clinicians identified core domains for use in clinical trials in SSc-associated RP and DUs, with several domains common to both manifestations of disease. These results will inform development of a final Core Domain Set for use in clinical trials.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/identifying-core-domains-for-clinical-trials-in-systemic-sclerosis-associated-raynauds-phenomenon-and-digital-ulcers-using-the-delphi-consensus-method/