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

Annemarie Schorpion, Max Shenin, Robin Neubauer, Chris T Derk, Annemarie Schorpion, Max Shenin, Robin Neubauer, Chris T Derk

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.

Keywords: Ambrisentan; Combination therapy; Endothelin; Endothelin receptor blocker; Mycophenolic mofetil; Open label; Scleroderma; Systemic sclerosis; Therapy; Treatment.

Conflict of interest statement

Study approved by the Institutional Review Board of the University of Pennsylvania and this was re-approved yearly during the study period. All patients who participated had to sign an informed consent approved by the Institutional Review Board of the University of Pennsylvania.Schorpion A, Shenin M, Neubauer R: No conflicts. Derk CT: Received research funding from Gilead.Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Figures

Fig. 1
Fig. 1
Study recruitment flowchart
Fig. 2
Fig. 2
Individual patient modified Rodnan skin scores at 0, 6 and 12 months of the study
Fig. 3
Fig. 3
Pulmonary Function Tests (PFTs) at 0, 6 and 12 months since initiation of ambrisentan

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