Optimizing Patient-Reported and Vascular Outcome Measures in Systemic Sclerosis-Associated Raynaud Phenomenon

ABSTRACT

In patient surveys, Raynaud Phenomenon (RP) is the highest-ranked disease-specific symptom affecting patient quality of life. Many promising vasoactive classes of drugs have been evaluated for RP, but none have received FDA approval for this indication. This project is designed to address four critical knowledge gaps that may influence clinical trial design: 1) What is the soundest approach to measure RP attack frequency/severity? Is the paper diary or a new smart phone application more functional and preferred? 2) What should be used as the patient- reported outcome (PRO)? 3) What is the magnitude of the effect of seasonal change on RP activity and severity? 4) What is the best provocation test to study vascular reactivity in early phase and proof-of-concept RP trials? We will use a prospective, observational cohort study design of 160 SSc-RP patients followed every 3 months over one year to accomplish all Specific Aims. Simultaneously, we will recruit 500 patients for a cross-sectional questionnaire study so that we may perform item response theory analysis and validation of the new ASRAP PRO.