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Abstract
BackgroundSystemic Sclerosis is a multifactorial autoimmune rheumatic disease characterized by inflammation, fibrosis, immune dysregulation and vascular dysfunction.MethodsAn open label, prospective, non-comparative study evaluating ambrisentan with an antifibrotic agent in diffuse cutaneous systemic sclerosis (dcSSc). Recruited 15 consecutive patients with dcSSc who were already on a stable dose of an antifibrotic agent and if they met inclusion criteria they were initiated on ambrisentan 5 mg/day for 12 months. Primary outcome measure was the modified Rodnan skin score (mRSS) while secondary measures were the short form 36 (SF-36) questionnaire, the Medsger severity score and pulmonary function studies.ResultsFifteen patients were recruited and ten patients completed all 12 months of the study. An intention to treat was used to analyze the data. There was statistical improvement of the mean mRSS and the perceived change in health component of the SF-36. The Medsger severity score and pulmonary function studies remained unchanged over the course of the study.ConclusionPatients who tolerated the combination of an antifibrotic with ambrisentan had an improvement of their mRSS over the course of the study as well as an improvement of their perceived health.Trial registrationClinicaltrials.gov, NCT01093885; March 2010.
Highlights
Systemic Sclerosis is a multifactorial autoimmune rheumatic disease characterized by inflammation, fibrosis, immune dysregulation and vascular dysfunction
On endothelial cells it leads to increased proliferation and increased vascular permeability, while on fibroblasts it leads to increased cell proliferation, increased expression of α- smooth muscle actin, and transformation of fibroblasts to myofibroblasts with increased extracellular matrix deposition and decreased fibroblast apoptosis
Patients who tolerated ambrisentan in combination with an antifibrotic agent showed an overall improvement in their modified Rodnan skin score (mRSS) and their perceived physical health, while over the 12 months of the study they did not have progression of their disease severity based on the Medsger severity scale
Summary
Systemic Sclerosis is a multifactorial autoimmune rheumatic disease characterized by inflammation, fibrosis, immune dysregulation and vascular dysfunction. It is believed that endothelial injury is the initial event which leads to vessel wall abnormality and an upregulation of adhesion molecules that in turn attract inflammatory cells which transmigrate through the Schorpion et al BMC Rheumatology (2018) 2:13 other extracellular matrix proteins that deposit in the vessel wall, and a subsequent transformation of fibroblasts into myofibroblasts. This results in functional and structural abnormalities of the vessel which in turn is theorized to cause similar changes to the organs that these vessels supply, such as the cutaneous tissue and the visceral organs [2,3,4]. Open label single center studies while inferior to the design of randomized controlled studies have in this particular disease helped establish certain agents as potential therapeutic agents in systemic sclerosis
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