A prospective, open-label, non-comparative study of ambrisentan with anti-fibrotic agent combination therapy in the treatment of diffuse systemic sclerosis

• Main Authors: Annemarie Schorpion, Max Shenin, Robin Neubauer, Chris T. Derk
• Format: Article
• Language: English
• Published: BMC 2018-05-01
• Series: BMC Rheumatology
• Subjects: Systemic sclerosis; Scleroderma; Ambrisentan; Combination therapy; Therapy; Mycophenolic mofetil
• Online Access: http://link.springer.com/article/10.1186/s41927-018-0021-z

Abstract Background Systemic Sclerosis is a multifactorial autoimmune rheumatic disease characterized by inflammation, fibrosis, immune dysregulation and vascular dysfunction. Methods An 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. Results Fifteen 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. Conclusion Patients 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 registration Clinicaltrials.gov, NCT01093885; March 2010.