Co-chairs: Mandy Nikpour (Melbourne) and Murray Baron (Montreal) SCTC Annual Business Meeting, Saturday November 15 th 2014

 Develop a disease damage index to systematically quantify organ damage in SSc. Potential applications: ◦ End-point in observational and interventional studies ◦ Quantifying disease burden in epidemiologic studies ◦ Describing differences between cohorts ◦ Enriching studies

 25 international members  Steering committee comprised of 7 of these members ◦ Mandy Nikpour, Murray Baron ◦ Marie Hudson, Susanna Proudman, Wendy Stevens ◦ Research Fellows: Tien Tay and Nava Ferdowsi (Melbourne)  Expert panel of non-rheumatologist advisors with a special interest in SSc  Methodologists/biostatisticians

 3 patient partners representing USA, UK and Australia ◦ Conceptual definition of damage ◦ Items included in the SSc-DI survey ◦ Reduced/final list of items

 First Working Group meeting at ACR ASM 2013  Two-round survey to obtain consensus regarding the conceptual definition of damage in SSc  Systematic review of existing measures of disease status in SSc  Item generation  Online survey hosted on REDCap currently open

 Permanent and irreversible loss of anatomical structure and physiologic function  Directly caused by SSc or its treatment and not due to comorbidities  Differentiated from disease activity (potentially reversible) and severity (no distinction between damage and activity)  PRO not included  Predictive of morbidity and/or mortality  Duration that an item must be present before being called damage depends on the item

 Based on systematic review  Input from expert advisors  Input from Working Group members ◦ Total of 83 items in 7 domains 1.Musculoskeletal and skin (19 items) 2.Vascular system (8 items) 3.Cardiac system (20 items) 4.Respiratory system (6 items) 5.Gastrointestinal system (17 items) 6.Renal system (4 items) 7.Damage from treatment (9 items)

 Each item rated according to its appropriateness (importance and feasibility) for inclusion in the damage index.  Duration that an item must be present before constituting damage also addressed  One round only

 69 complete responses to date  Further 12 as yet incomplete responses respondents required to power the planned statistical analyses to reduce the items. All respondents acknowledged in publications.

Asia n=1 Australia = 15 Europe = 34 North America = 16 South America = 3

 Rasch modeling to reduce the items  Structural equation modeling (SEM) to weight the items against mortality using Australian (and Canadian) cohort data

 L201 – 2 nd floor School of Medicine (L- Building) at 72E ◦ Further discussion of points arising from preliminary analysis of survey data ◦ Please address enquiries to Mandy Nikpour Murray Baron

Please respond to the survey if you have not done so already. The success of this project depends on your response.