Identifying candidate core domains for clinical trials in systemic sclerosis-associated Raynaud's phenomenon and digital ulcers.

BACKGROUND

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/or digital ulcers (DUs) related to systemic sclerosis (SSc).

METHODS

Candidate domains identified from previous qualitative work and systematic literature reviews were included in separate Delphi exercises for SSc-RP and SSc-DUs. Patients with SSc and other participants (clinicians with experience in treating patients with SSc and/or conducting clinical trials) were invited to participate through international patient advocacy groups and clinical networks. Core domains were defined as the domains reaching group consensus agreement (≥ 70% ratings of 'critical' in both patient and other participant groups) after three rounds of the Delphi.

RESULTS

The 3-round Delphi exercises were completed by 78 patients and 116 others from 39 countries for SSc-RP, and by 26 patients and 99 others from 36 countries for SSc-DUs. For SSc-RP, nine domains reached consensus agreement as critically important: three domains in the Pathophysiological Manifestations core area and six in the Life Impact core area. For SSc-DUs, 16 domains reached consensus: five domains in the Pathophysiological Manifestations core area, seven in the Life Impact core area, and four in the Resource Use area.

CONCLUSION

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