Children's Hospital at Montefiore new york, NY, United States
Natalia Vasquez Canizares1, Clare Pain2, Marinka Twilt3, Amra Adrovic Yildiz4, Simone Appenzeller5, Edoardo Marrani6, Emanuela Del Giudice7, Antonella Petaccia8, Francesca Tirelli9, Gabriele Simonini10, Marco Cattalini11, Ozgur Kasapcopur12, Raju Khubchandani13, Kathryn Torok14, Betul Sozeri15, Biagio Castaldi16, Katherine Clarke17, Phuoc Duong17, Aybuke Gunalp18, meiping lu19, Hanna Lythgoe2, Amanda Robinson14, Lauren Robinson20, Paulo Rogerio Julio21, Susan Shenoi22, Emily Willis23, Mustafa Çakan15, Valentina Leone24, Rongjun Zheng19, Phillip Lawrence17, Franziska Rosser25, Sarah Mayell2, Lusine Ambartsumyan26, Aurelia Minuti27, Francesco Zulian28 and Suzanne Li29, 1Children's Hospital at Montefiore/ Albert Einstein College of Medicine, New York, NY, 2Alder Hey Children’s NHS Foundation Trust, Liverpool, United Kingdom, 3Alberta Children's Hospital, Calgary, AB, Canada, 4School of Medicine, KOC University, Istanbul, Turkey, 5Unicamp, Campinas, São Paulo, Brazil, 6AOU Meyer, Florence, Italy, 7Sapienza University of Rome, Pediatric and Neonatology Unit, Maternal and Child Department, Rome, Italy, 8Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy, 9Women's and Child's Health, University Hospital of Padova, Padova, Italy, 10Meyer Children's Hospital, Florence, Italy, 11Pediatric Clinic, University of Brescia, Brescia, Italy, 12Istanbul University-Cerrahpaşa, Cerrahpaşa Medical School, Istanbul, Turkey, 13SRCC Children's Hospital, Mumbai, India, 14Pediatric Rheumatology, Children's Hospital of UPMC, Pittsburgh, PA, 15University of Health Sciences, Istanbul, Turkey, 16Women's and Child's Health, University Hospital of Padova, Padua, Italy, 17Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom, 18Istanbul University Cerrahpaşa Medical School, Istanbul, Turkey, 19Children’s Hospital of Zhejiang University Medical School, Zhejiang, China, 20Hospital for Special Surgery, New York, NY, 21University of Campinas, Campinas, São Paulo, Brazil, 22Seattle Children's Hospital and Research Center / University of Washington, Seattle, WA, 23Royal Manchester Children’s Hospital, Manchester, United Kingdom, 24Ismett, UPMC, Palermo, Italy, 25University of Pittsburgh, Children’s Hospital of UPMC, Pittsburgh, PA, 26Seattle Children's Hospital, University of Washington, Seattle, WA, 27D. Samuel Gottesman Library Research and Education, Albert Einstein College of Medicine, Bronx, NY, 28University of Padua, Padua, Italy, 29Hackensack Meridian School of Medicine, Joseph M. Sanzari Children's Hospital, West Orange, NJ
Background/Purpose: Juvenile Systemic Sclerosis (jSSc) is an autoimmune and fibrosing disease associated with significant morbidity and mortality risk. Data on treatment is limited and based mainly on adult data despite the many differences between adult and jSSc. There is a need for international jSSc consensus tools and definitions in order to conduct jSSc treatment studies
Methods: An international effort primarily between pediatric rheumatologists (PRs) in CARRA and the Pediatric Rheumatology European Society (PRES) is being led by a core group of 3 CARRA and 2 PRES members (NV, SL, MT, CP, FZ). These steps are planned: literature review (LR) on SSc measures, development of surveys to understand current management strategies, and Delphi consensus meetings. The aim of the LR is to identify available measures used in SSc including adults, and their properties for each of 6 areas (cardiac, pulmonary, vascular, gastrointestinal, skin/musculoskeletal, or health-related quality of life). Results will be used to inform the development of surveys evaluating the use and feasibility of these measures, and scoring levels used by PRs. Covidence is being used to facilitate the LR process
Results: We conducted an initial survey of CARRA and a subset of PRES members to understand the prevalence of jSSc, most commonly used assessments and treatments for cardiopulmonary (CP) disease, and identify PRs interested in participating in our study. Response rate was 25% from 348 CARRA, and 35% from 156 PRES members. Responders from 26 countries follow 649 jSSc patients, with a median of 3 patients followed per center (IQR 2-7). Frequency use of CP assessment tools and proportion of most frequently used immunosuppressive treatments by PRs are shown in Table 1 and Figure 1. The pulmonary function test measurement considered most important for lung evaluation was the DLCO (75%), whereas echocardiogram (77%) was considered the most important cardiac tool. The 3 most commonly used treatments were oral steroids, methotrexate, and mycophenolate mofetil, each used in about 20% of patients. For our LR, 24,849 unique articles identified from 4 databases were screened by 18 PRs. Inclusion criteria included English language, original peer-reviewed research related to SSc, and including a measure from 1 of the 6 areas of interest. A total of 21,565 articles were eliminated, leaving 3284 articles. These articles have been screened by 34 collaborators for relevance by each of the 6 organ-focused groups. From this screen, 300-600 references remain in each organ group for full text screening to identify articles relevant for data extraction
Conclusion: We have established collaboration with 34 members from 12 countries who will be participating in this effort. Our initial survey of 141 PRs identified variation in preferences for CP assessments and treatments. A high proportion of PRs indicated that they have never used or do not have access to some cardiac assessment tools and biologics to manage their jSSc patients. We completed the first phase of our LR, identifying 300-600 articles in each of the 6 organ-focused groups for further screening followed by data extraction. This effort will generate consensus on outcome measures and definitions to use in future jSSc treatment trials
Disclosures: N. Vasquez Canizares, None; C. Pain, None; M. Twilt, None; A. Adrovic Yildiz, None; S. Appenzeller, None; E. Marrani, None; E. Del Giudice, None; A. Petaccia, None; F. Tirelli, None; G. Simonini, None; M. Cattalini, None; O. Kasapcopur, None; R. Khubchandani, None; K. Torok, None; B. Sozeri, None; B. Castaldi, None; K. Clarke, None; P. Duong, None; A. Gunalp, None; m. lu, None; H. Lythgoe, None; A. Robinson, None; L. Robinson, None; P. Julio, None; S. Shenoi, None; E. Willis, None; M. Çakan, None; V. Leone, None; R. Zheng, None; P. Lawrence, None; F. Rosser, None; S. Mayell, None; L. Ambartsumyan, None; A. Minuti, None; F. Zulian, None; S. Li, None.
