Suzanne C Li1,2, Xiaohu Li3,4, Elena Pope3,4, Katie Stewart3,4, Gloria C Higgins3,4, C Egla Rabinovich3,4, Kathleen M O'Neil3,4, Kathleen A Haines3,4, Ronald M Laxer3,4, Marilynn Punaro3,4, Heidi Jacobe3,4, Tracy Andrews3,4, Knut Wittkowski3,4, Themba Nyirenda3,4, Ivan Foeldvari3,4, Kathryn S Torok3,4. 1. From the Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics, Hackensack; Stevens Institute of Technology, Hoboken, New Jersey, USA; Hospital for Sick Children, Toronto, Ontario, Canada; Texas Scottish Rite Hospital, University of Texas (UT) Southwestern, Dallas, Texas; The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio; Duke University, Durham, North Carolina; Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana; Rockefeller University, New York, New York, USA; Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA. suzanne.li@hackensackmeridian.org. 2. S.C. Li, MD, PhD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics; X. Li, PhD, Stevens Institute of Technology; E. Pope, MD, Hospital for Sick Children; K. Stewart, MD, Texas Scottish Rite Hospital, UT Southwestern; G.C. Higgins, MD, PhD, The Ohio State University and Nationwide Children's Hospital; C.E. Rabinovich, MD, MPH, Duke University; K.M. O'Neil, MD, Riley Hospital for Children at Indiana University Health; K.A. Haines, MD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics; R.M. Laxer, MD, Hospital for Sick Children; M. Punaro, MD, Texas Scottish Rite Hospital, UT Southwestern; H. Jacobe, MD, UT Southwestern; T. Andrews, MA, Hackensack University Medical Center; K. Wittkowski, PhD, Rockefeller University; T. Nyirenda, PhD, Hackensack University Medical Center; I. Foeldvari, MD, Hamburger Zentrum für Kinder- und Jugendrheumatologie; K.S. Torok, MD, Children's Hospital of Pittsburgh. suzanne.li@hackensackmeridian.org. 3. From the Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics, Hackensack; Stevens Institute of Technology, Hoboken, New Jersey, USA; Hospital for Sick Children, Toronto, Ontario, Canada; Texas Scottish Rite Hospital, University of Texas (UT) Southwestern, Dallas, Texas; The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio; Duke University, Durham, North Carolina; Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana; Rockefeller University, New York, New York, USA; Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA. 4. S.C. Li, MD, PhD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics; X. Li, PhD, Stevens Institute of Technology; E. Pope, MD, Hospital for Sick Children; K. Stewart, MD, Texas Scottish Rite Hospital, UT Southwestern; G.C. Higgins, MD, PhD, The Ohio State University and Nationwide Children's Hospital; C.E. Rabinovich, MD, MPH, Duke University; K.M. O'Neil, MD, Riley Hospital for Children at Indiana University Health; K.A. Haines, MD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics; R.M. Laxer, MD, Hospital for Sick Children; M. Punaro, MD, Texas Scottish Rite Hospital, UT Southwestern; H. Jacobe, MD, UT Southwestern; T. Andrews, MA, Hackensack University Medical Center; K. Wittkowski, PhD, Rockefeller University; T. Nyirenda, PhD, Hackensack University Medical Center; I. Foeldvari, MD, Hamburger Zentrum für Kinder- und Jugendrheumatologie; K.S. Torok, MD, Children's Hospital of Pittsburgh.
Abstract
OBJECTIVE: To identify clinical features that define disease activity in pediatric localized scleroderma (LS), and determine their specificity and importance. METHODS: We conducted a multicenter prospective study of patients with active and inactive LS skin lesions. A standardized evaluation of a single designated study lesion per subject was performed at 3 visits. We evaluated the pattern and correlation between assessed features and physician's global assessments of activity (PGA-A). RESULTS: Ninety of 103 subjects had evaluable data; 66 had active and 24 inactive disease. Subjects had similar age of onset, sex, and disease patterns. Linear scleroderma was the most common subtype. Features specific for active disease included erythema, violaceous color, tactile warmth, abnormal skin texture, and disease extension. Scores for these variables changed over time and correlated with PGA-A of the lesion. Active and inactive lesions could not be distinguished by the presence or level of skin thickening, either of lesion edge or center. However, in active lesions, skin thickening scores did correlate with PGA-A scores. Regression analysis identified the combination of erythema, disease extension, violaceous color, skin thickening, and abnormal texture as predictive of PGA-A at study entry. Damage features were common irrespective of activity status. CONCLUSION: We identified variables strongly associated with disease activity, expanding upon those used in current measures, and determined their relative importance in physician activity scoring. Skin thickening was found to lack specificity for disease activity. These results will help guide development of a sensitive, responsive activity tool to improve care of patients with LS.
OBJECTIVE: To identify clinical features that define disease activity in pediatric localized scleroderma (LS), and determine their specificity and importance. METHODS: We conducted a multicenter prospective study of patients with active and inactive LS skin lesions. A standardized evaluation of a single designated study lesion per subject was performed at 3 visits. We evaluated the pattern and correlation between assessed features and physician's global assessments of activity (PGA-A). RESULTS: Ninety of 103 subjects had evaluable data; 66 had active and 24 inactive disease. Subjects had similar age of onset, sex, and disease patterns. Linear scleroderma was the most common subtype. Features specific for active disease included erythema, violaceous color, tactile warmth, abnormal skin texture, and disease extension. Scores for these variables changed over time and correlated with PGA-A of the lesion. Active and inactive lesions could not be distinguished by the presence or level of skin thickening, either of lesion edge or center. However, in active lesions, skin thickening scores did correlate with PGA-A scores. Regression analysis identified the combination of erythema, disease extension, violaceous color, skin thickening, and abnormal texture as predictive of PGA-A at study entry. Damage features were common irrespective of activity status. CONCLUSION: We identified variables strongly associated with disease activity, expanding upon those used in current measures, and determined their relative importance in physician activity scoring. Skin thickening was found to lack specificity for disease activity. These results will help guide development of a sensitive, responsive activity tool to improve care of patients with LS.
Entities:
Keywords:
DISEASE ACTIVITY; LOCALIZED SCLERODERMA; PHYSICIAN’S GLOBAL ASSESSMENT
Authors: Kathryn S Torok; Suzanne C Li; Heidi M Jacobe; Sarah F Taber; Anne M Stevens; Francesco Zulian; Theresa T Lu Journal: Front Immunol Date: 2019-04-30 Impact factor: 7.561
Authors: Suzanne C Li; Robert C Fuhlbrigge; Ronald M Laxer; Elena Pope; Maria F Ibarra; Katie Stewart; Thomas Mason; Mara L Becker; Sandy Hong; Fatma Dedeoglu; Kathryn S Torok; C Egla Rabinovich; Polly J Ferguson; Marilynn Punaro; Brian M Feldman; Tracy Andrews; Gloria C Higgins Journal: Pediatr Rheumatol Online J Date: 2019-07-15 Impact factor: 3.054