{ "abstract": "Introduction Limited cutaneous Systemic sclerosis (lcSSc) and sine scleroderma constitute more than 60% of all SSc patients. There is a lack of high quality randomized controlled trials conducted for lcSSc. Acquiring high quality data on the natural history of early lcSSc will help design trials with a higher probability of success. CONQUER is a multicenter US-based cohort of patients with early SSc. This analysis of CONQUER aimed to describe the prevalence of organ involvement in lcSSc patients and to define the onset of key scleroderma manifestations during follow-up. Baseline characteristics predicting disease progression were also identified.Material and Methods All patients from the CONQUER multicenter US cohort with physician diagnosed sine scleroderma or lcSSc at baseline were included. Patients were defined as progressors if they experienced one or more of the following events: New renal crisis, Interstitial Lung disease (ILD) progression (relative loss of more than 15% of FVC as compared to first available FVC or onset of FVC less than 80% with ILD), onset of heart failure (LVEF bellow 40%), pulmonary arterial hypertension on right heart catheterization, gastrointestinal dysmotility requiring enteral/parenteral nutrition, digital ulcer (DU) or gangrene, or death.Results 275 patients were included, with a mean (SD) disease duration of 2.6 (1.39) years since first non-RP and 6.2 (8.51) years since RP. Baseline manifestations included: active digital ulcers in 29.5%, ILD in 34.9%, SSc-related cardiac involvement in 12.4% and renal crisis in 1.5%. Only 14 patients (5%) were identified as progressors in the first 12 month of follow-up. Twenty-nine patients (11%) were identified as progressors in the first 24 months of follow-up. The main reasons for progression at 24 months were ILD progression (55.2%) and new DU (17.2%) during follow-up. Baseline characteristics predicting progression were positivity for anti-topoisomerase-1 antibody (antiTOPO) compared to anti centromere (OR 3.20, 95%CI(1.17-9.65)), the presence of ILD (OR 4.10, 95%CI(1.45-14.69)), the presence of dyspnea (NYHA II, III or IV) (OR 3.05, 95%CI(1.38-7.01), FVC (%pred) less than 80% (OR 2.44, 95%CI(1.06-5.46)), and DLCO (%pred) less than 80% (OR 3.26, 95%CI(1.42-8.15)).Conclusions In the CONQUER database of patients with early SSc, 5% and 11% of patients with lcSSc or sine scleroderma experienced disease progression at 12 and 24 months, respectively. This low rate of progression suggests that clinical trials may benefit from a trial duration of 24 months. Incorporating lcSSc patients with antiTOPO and presence of ILD may help enrich clinical trials with lcSSc patients more likely to show disease progression.", "authors": [ { "affiliations": [ "Scleroderma programm, Rennes University, CHU Rennes, Rennes, France", "Division of Rheumatology, Department of Medicine, University of Michigan, Ann Arbor, USA" ], "name": "Alain Lescoat" }, { "affiliations": [ "Department of Medicine, Georgetown University Medical Center, Washington, USA" ], "name": "Virginia Steen" }, { "affiliations": [ "Division of Pediatric Critical Care, Department of Pediatrics, University of Utah, Salt Lake City, USA" ], "name": "Monica Harding" }, { "affiliations": [ "Division of Pediatric Critical Care, Department of Pediatrics, University of Utah, Salt Lake City, USA" ], "name": "John M VanBuren" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Texas Health Science Center at Houston, Houston, USA" ], "name": "Brian Skaug" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Texas Health Science Center at Houston, Houston, USA" ], "name": "Shervin Assassi" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Texas Health Science Center at Houston, Houston, USA" ], "name": "Maureen D Mayes" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Texas Health Science Center at Houston, Houston, USA" ], "name": "Zsuzsanna H McMahan" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Columbia University Irving Medical Center, New York, USA" ], "name": "Elana J Bernstein" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Massachusetts General Hospital, Boston, USA" ], "name": "Flavia V Castelino" }, { "affiliations": [ "Division of Immunology and Rheumatology, Department of Medicine, Stanford University, Palo Alto, USA" ], "name": "Lorinda Chung" }, { "affiliations": [ "Scleroderma Research Foundation, San Francisco, USA" ], "name": "Luke B Evnin" }, { "affiliations": [ "Division of Rheumatology and Immunology, Department of Medicine, Vanderbilt University Medical Center, Nashville, USA" ], "name": "Tracy M Frech" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Hospital for Special Surgery, New York, USA" ], "name": "Jessica K Gordon" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Medical University of South Carolina, Charleston, USA" ], "name": "Faye N Hant" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Johns Hopkins University, Baltimore, USA" ], "name": "Laura K Hummers" }, { "affiliations": [ "Division of Rheumatology, Department of Internal Medicine, University of Utah, Salt Lake City, USA" ], "name": "Kimberly S Lakin" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Hospital for Special Surgery, New York, USA" ], "name": "Dorota Lebiedz-Odrobina" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Columbia University Irving Medical Center, New York, USA" ], "name": "Yiming Luo" }, { "affiliations": [ "Division of Rheumatology, Department of Internal Medicine, Mayo Clinic, Rochester, USA" ], "name": "Ashima Makol" }, { "affiliations": [ "Division of Rheumatic and Autoimmune Diseases, Department of Medicine, University of Minnesota, Minneapolis, USA" ], "name": "Jerry A Molitor" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Northwestern University, Chicago, USA" ], "name": "Duncan F Moore" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Northwestern University, Chicago, USA" ], "name": "Carrie Richardson" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Pennsylvania, Philadelphia, USA" ], "name": "Nora Sandorfi" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, Johns Hopkins University, Baltimore, USA" ], "name": "Ami A Shah" }, { "affiliations": [ "Division of Rheumatology and Immunology, Department of Medicine, Duke University, Durham, USA" ], "name": "Ankoor Shah" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, USA" ], "name": "Elisabeth R Volkmann" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Michigan, Ann Arbor, USA" ], "name": "Carleigh Zahn" }, { "affiliations": [ "Division of Rheumatology, Department of Medicine, University of Michigan, Ann Arbor, USA" ], "name": "Dinesh Khanna" } ], "title": "OC.25 Prevalence of organ involvement and baseline predictors of disease progression in patients with limited cutaneous systemic sclerosis: insights from the conquer database", "uid": "e9ad5135-4d78-5d13-8cf5-b617921f4601" }