“Effect of Macitentan on the Development of New Ischemic Digital Ulcers in Patients With Systemic Sclerosis: DUAL-1 and DUAL-2 Randomized Clinical Trials”
The Journal of the American Medical Association, 2016
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Dr. Peter Merkel* |
- DUAL1 and 2 were two similar large trials run in parallel to test a new treatment for digital ulcers in patients with systemic sclerosis (scleroderma). This was the largest research program ever conducted to study digital ulcers in scleroderma.
- Unfortunately, these trials did not show any benefit of the study drug (macitentan) for treating digital ulcers. However, the study was well done and this helps physicians and patients avoid using drugs that are not effective.
- Additional analysis of the data from these trials are now ongoing and will help us understand digital ulcers better.
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“Standardization of the Modified Rodnan Skin Score for Use in Clinical Trials of Systemic Sclerosis”
SAGE Journals, 2017
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Dr. Peter Merkel |
- This paper outlines a standard way for all physicians to measure the degree of skin disease in patients with scleroderma so that clinical trials can be conducted in a standardized fashion leading to better quality data.
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“Measuring disease activity and functional status in patients with scleroderma and Raynaud's phenomenon”
Arthritis & Rheumatology, 2002
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Dr. Peter Merkel* |
- This project validated a new measure of Raynaud's phenomenon that has been used in many clinical trials.
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“Following the molecular pathways toward an understanding of the pathogenesis of systemic sclerosis”
Ann Intern Med, 2004
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Dr. Chris Derk
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- A comprehensive review of the molecular pathophysiology of systemic sclerosis.
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“The American College of Rheumatology Provisional Composite Response Index for Clinical Trials in Early Diffuse Cutaneous Systemic Sclerosis”
Arthritis & Rheumatology, 2016
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Dr. Peter Merkel
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- This project used data collected over several years from patients with scleroderma to create a new way to measure the level of disease in scleroderma to help conduct clinical trials in a new way.
- This new tool for researchers (CRISS) was endorsed by the The American College of Rheumatology and is now in regular use in clinical trials.
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“Assessment of skin disease in scleroderma: Practices and opinions of investigators studying scleroderma”
SAGE Journals, 2020
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Dr. Peter Merkel |
- This study assessed how physicians studying scleroderma around the world measure skin disease and offers insight into how to improve this critical aspect of research in scleroderma.
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“A cohort study of cancer incidence in systemic sclerosis”
J Rheumatol, 2006
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Dr. Chris Derk*
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- A single center analysis of cancer incidence in systemic sclerosis patients
- Increase in the incidence of cancer among systemic sclerosis patients compared to the general population and specifically esophageal and oropharyngeal cancers.
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“A prospective open-label study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis”
Rheumatology, 2009
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Dr. Chris Derk* |
- One of the first open label studies looking at mycophenolate mofetil for the treatment of diffuse systemic sclerosis
- Improvement of skin scores, peripheral vascular involvement and patient-perceived health status as 52 weeks of therapy.
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“ISHLT consensus document on lung transplantation in patients with connective tissue disease: Part I: Epidemiology, assessment of extrapulmonary conditions, candidate evaluation, selection criteria, and pathology statements”
J Heart Lung Transplant, 2021
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Dr. Nora Sandorfi* |
- This important publication was led by members of the Penn Scleroderma Center and outlines the process for evaluating patients with scleroderma for possible lung transplantation. Penn Medicine is a leading worldwide center for organ transplantation.
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“Gastric antral vascular ectasia in systemic sclerosis: demographics and disease predictors"
J Rheumatol, 2010
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Dr. Chris Derk |
- A two-center analysis of disease predictors in the development of gastric antral vascular ectasia (GAVE) in systemic sclerosis.
- Diffuse systemic sclerosis is associated with earlier development of GAVE, as well as more severe anemia. Absence of anti-topoisomerase I antibody and presence of RNA Poly III antibodies possible predictors of GAVE.
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“Mortality, Recurrence, and hospital course of patients with systemic sclerosis-related acute intestinal pseudo-obstruction”
J Rheumatol, 2014
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Dr. Nora Sandorfi
Dr. Chris Derk
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- A two-center analysis and hospital course of systemic sclerosis patients with intestinal pseudoobstruction.
- Patients with recurrent pseudoobstruction in systemic sclerosis were more likely to require prolonged TPN, mortality was higher in male patients.
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“Mortality, length of stay and cost of hospitalization among patients with systemic sclerosis: results from the National Inpatient Sample”
Rheumatology (Oxford, England), 2018
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Dr. Nora Sandorfi
Dr. Chris Derk
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- National inpatient sample analysis of in hospital mortality of patients with systemic sclerosis.
- Infections were the most common reason for admission and in hospital death in systemic sclerosis patients.
- This study used national data to document the reasons patients with scleroderma are admitted to the hospital and highlighted the critical role of infections among patients with scleroderma
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“Abatacept in Early Diffuse Cutaneous Systemic Sclerosis: Results of a Phase II Investigator-Initiated, Multicenter, Double-Blind, Randomized, Placebo-Controlled Trial”
Arthritis & Rheumatology, 2020
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Dr. Nora Sandorfi |
- This was a multi-center clinical trial tested a biologic drug for the treatment of scleroderma.
- It is extremely important that the scleroderma research community continues to work together to advance research in this complex disease.
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“Risk factors for mortality and cardiopulmonary hospitalization in systemic sclerosis patients at risk for pulmonary hypertension, in the PHAROS registry”
J Rheumatol, 2019
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Dr. Chris Derk |
- A multicenter, long term data analysis of the PHAROS database looking at predictors of mortality and hospitalization in systemic sclerosis patients with pulmonary hypertension
- Risk factors for mortality in systemic sclerosis patients with pulmonary hypertension were male sex, a DLC<50%, exercise oxygen desaturation and pericardial effusions
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“HLA and autoantibodies define scleroderma subtypes and risk in African and European Americans and suggest a role for molecular mimicry”
Proc Natl Acad Sci, 2020
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Dr. Chris Derk |
- A multicenter NIH sponsored study looking at HLA and autoantibodies in different subtypes of systemic sclerosis among African and European Americans
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“Avascular necrosis in systemic sclerosis patients: a case-based review of demographics, presentation, and management”
Clin Rheumatol, 2020
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Dr. Chris Derk |
- A case-based review of the literature of the predictors of avascular necrosis in systemic sclerosis patients
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