Clinical characteristics, visceral involvement, and mortality in at-risk or early diffuse systemic sclerosis: a longitudinal analysis of an observational prospective multicenter US cohort

Arthritis Research & Therapy

Elana J. Bernstein¹  Victoria K. Shanmugam²  Virginia Steen³  Sabrina Elliott³  Flavia V. Castelino⁴  Jessica Gordon⁵  Ami A. Shah⁶  Faye Hant⁷  Monique Hinchcliff⁸  Chase Correia⁸  Robyn Domsic⁹  Shervin Assassi^1,10  Tracy Frech^1,11  Vivek Nagaraja^1,12  David Roofeh^1,12  Sara Jaafar^1,12  Dinesh Khanna^1,12  John Varga^1,12  Alain Lescoat^1,13  Suiyuan Huang^1,14 

[1]Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA

[2]Department of Medicine, George Washington University, Washington, DC, USA

[3]Department of Medicine, Georgetown University, Washington, DC, USA

[4]Department of Medicine, Harvard University, Boston, MA, USA

[5]Department of Medicine, Hospital for Special Surgery, New York, NY, USA

[6]Department of Medicine, Johns Hopkins University, Baltimore, MD, USA

[7]Department of Medicine, Medical University of South Carolina, Charleston, SC, USA

[8]Department of Medicine, Northwestern University, Chicago, IL, USA

[9]Department of Medicine, University of Pittsburgh Medical Center, Pittsburgh, PA, USA

[10]Department of Medicine, University of Texas Health Science Center, Houston, TX, USA

[11]Department of Medicine, University of Utah, Salt Lake City, UT, USA

[12]Division of Rheumatology and Scleroderma Program, Department of Internal Medicine, University of Michigan, Suite 7C27 300 North Ingalls Street, SPC 5422, 48109, Ann Arbor, MI, USA

[13]Division of Rheumatology and Scleroderma Program, Department of Internal Medicine, University of Michigan, Suite 7C27 300 North Ingalls Street, SPC 5422, 48109, Ann Arbor, MI, USA

[14]Department of Internal Medicine and Clinical Immunology, CHU Rennes, Univ Rennes, Rennes, France

[15]University of Rennes, CHU Rennes, Inserm, EHESP, Irset (Institut de recherche en santé, environnement et travail, Rennes, France

[16]School of Public Health, University of Michigan, Ann Arbor, USA

关键词: Systemic sclerosis;    Scleroderma;    Diffuse cutaneous systemic sclerosis;    Mortality;    Survival;    Interstitial lung disease;   

DOI  :  10.1186/s13075-021-02548-1

来源: Springer

【 摘 要 】

BackgroundEarly diffuse cutaneous systemic sclerosis (dcSSc) has the highest case fatality among rheumatic diseases. We report baseline characteristics, current immunosuppressive therapies, progression of skin and internal organ involvement, and mortality in a multicenter prospective cohort from the United States (US) of America.MethodsWe performed a longitudinal analysis of participants from 12 US centers, from April 2012 to July 2020. All participants had early dcSSc or were at-risk for dcSSc, with ≤2 years since the first non-Raynaud’s phenomenon (RP) symptom.ResultsThree hundred one patients were included with a baseline median disease duration of 1.2 years since RP and a mean modified skin score of 21.1 units. At baseline, 263 (87.3%) had definite dcSSc and 38 (12.7%) were classified as at-risk; 112 (49.6%) patients were positive for anti-RNA polymerase III antibodies. The median follow-up duration was 24.5 months (IQR = 10.3–40.7 months). One hundred ninety (63.1%) participants were treated with an immunosuppressive therapy, of which mycophenolate mofetil was most used at baseline and follow-up. Of 38 who were classified as at-risk at baseline, 27 (71%) went on to develop dcSSc; these patients were characterized by higher baseline mean HAQ-DI (0.8 versus 0.4, p = 0.05) and higher baseline mRSS (8.8 versus 4.4, p < 0.01) in comparison with those who remained as limited cutaneous SSc. In the overall cohort, 48 participants (21.1%) had clinically significant worsening of skin fibrosis, mainly occurring in the first year of follow-up; 41 (23.3%) had an absolute forced vital capacity decline of ≥10%. Twenty participants (6.6%) died, of which 18 died in the first 3 years of follow-up. Cardiac involvement (33.3%), gastrointestinal dysmotility (22.2%), and progressive interstitial lung disease (ILD) (16.7%) were the main causes of death.ConclusionThis US cohort highlights the management of early SSc in the current era, demonstrating progression of skin and lung involvement despite immunosuppressive therapy and high mortality due to cardiac involvement.

【 授权许可】

CC BY