Author(s)
Domsic RT1, Vampola CL2, Stassek L3, Pokrzywinski R4, Furst D5, Chung LS6, Steen V7, Mayes MD8, Shah A9, Molitor J10, Nagaraja V11, Oliver K12, Benton W13, Khanna D11
1University of Pittsburgh Department of Medicine, Pittsburgh, PA, USA, 2Eicos Sciences, Chevy Chase, MD, USA, 3Evidera, Seattle, WA, USA, 4EVIDERA, Bethesda, MD, USA, 5UCLA, Los Angeles, CA, USA, 6Stanford University, Palo Alto, CA, USA, 7Georgetown University Medical Center, Washington, DC, USA, 8University of Texas, Houston, TX, USA, 9John Hopkins Hospital, Baltimore, MD, USA, 10University of Minnesota, Minneapolis, MN, USA, 11University of Michigan, Ann Arbor, MI, USA, 12Eicos Sciences, San Mateo, CA, USA, 13Eicos Sciences, Redwood City, CA, USA
OBJECTIVES: Systemic Sclerosis (SSc) is a rare, multisystem, autoimmune rheumatic disease with >95% of patients experiencing digital ischemic episodes (Raynaud phenomenon, RP) that impact quality of life (QOL). The objectives of this study were to understand the nature and impact of symptomatic RP in SSc patients and to evaluate comprehension, relevance, usability, and feasibility of a novel electronic patient-reported outcome measure (ePRO). METHODS: The study was granted an exemption by a Central IRB. Participants who met 2013 ACR/EULAR classification criteria for SSc and had symptomatic RP were recruited via flyers from two university clinics and local foundation chapters. An experienced moderator conducted 1-on-1, in-person, qualitative interviews facilitated by a semi-structured interview guide. Interview transcripts were analyzed using ATLAS.ti version 8.3.17. RESULTS: Twenty participants had mean(SD) age of 50.9(12.3) years and were further classified into diffuse cutaneous (n=11, 55%), limited cutaneous (n=6, 30%), overlap (n=2, 10%), or unknown SSc subtype (n=1, 5%). Most were female (n=16, 80%). All participants reported at least one finger color change (white/red/blue/purple) accompanied with symptoms of pain (reported by 95%), numbness (95%), discomfort (90%), and tingling (85%). Variability in attack triggers, duration, and frequency were noted within and between subjects. All participants (100%) stated RP impacted daily life, including avoiding/limiting outdoor activities and having difficulty gripping items. All participants gave positive feedback on comprehension and relevance of diary content. They all reported being comfortable with the ePRO device, finding it to be straightforward to use and noted it would be feasible to complete the daily diary for two weeks. CONCLUSIONS: RP has a significant negative impact on QOL in people with SSc. The current research provides face and content validity of a novel RP diary and supports its use in trials of symptomatic RP in patients with SSc.
Conference/Value in Health Info
2021-05, ISPOR 2021, Montreal, Canada
Value in Health, Volume 24, Issue 5, S1 (May 2021)
Code
PRO62
Topic
Clinical Outcomes, Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment, Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes, PRO & Related Methods
Disease
Rare and Orphan Diseases