A pilot trial of treprostinil for the treatment and prevention of digital ulcers in patients with systemic sclerosis
ABSTRACT We performed a pilot trial of subcutaneous treprostinil for the treatment of digital ulcers in scleroderma. Of the 5 patients completing therapy, ulcer size significantly decreased and no new ulcers occurred on continuous therapy. Although effective, the high rate of injection site reactions may limit the utility of this therapy.
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- "International Journal of Rheumatology subcutaneous delivery    "
ABSTRACT: Systemic sclerosis is a connective tissue disease characterized by fibrosis of the skin, internal organs, and widespread vasculopathy. Raynaud's phenomenon and digital ulcers are vascular manifestations of this disease and cause significant morbidity. Current treatments are only moderately effective in reducing the severity of Raynaud's in a portion of patients and typically do not lead to substantial benefit in terms of the healing or prevention of digital ulcers. Several studies have evaluated the efficacy of targeting the vasoconstrictor endothelin-1 for the treatment of systemic sclerosis-associated vascular disease. The purpose of this paper is to summarize the published studies and case reports evaluating the efficacy of endothelin receptor antagonists in the treatment of Raynaud's phenomenon and digital ulcers associated with systemic sclerosis.International Journal of Rheumatology 10/2011; 2011(2):201787. DOI:10.1155/2011/201787
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- "Treprostinil is available in IV, subcutaneous, inhaled, and oral formulations. A pilot study of subcutaneous treprostinil in patients with SSc-DUs over a 12-week treatment period showed both healing and prevention of the DUs but only a few subjects were able to tolerate the injection site pain . Treprostinil diethanolamine (TDE-SR) is an innovative salt form of treprostinil for oral delivery as a sustained release osmotic tablet for twice daily dosing. "
ABSTRACT: Systemic Sclerosis (Scleroderma, SSc) is a disease of unknown etiology characterized by widespread vasculopathy and extracellular matrix deposition leading to fibrosis and autoimmune processes. Digital ischemia (digital ulcers (DUs)) is the hallmark of SSc-related vasculopathy and is characterized by endothelial dysfunction leading to intimal proliferation and thrombosis. It happens frequently (30% of the patients each year) and it is associated with significant morbidity. This paper summarizes the current information regarding pathogenesis, definitions, management, and exploratory therapies in DUs associated with SSc.International Journal of Rheumatology 08/2010; 2010. DOI:10.1155/2010/923743
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ABSTRACT: Raynaud's phenomenon (RP) is characterised by transient ischaemia in the extremities in response to cold or emotions. It can be primary (idiopathic) or secondary to an underlying disease. The pathophysiology of RP is multifactorial and complex. Microvascular impairment is a hallmark of the disease. The objective of this work is to review the different pharmacological treatments currently used in the management of RP, from their mechanism of action to the available evidence regarding their efficacy. We also propose to discuss potential pharmacological targets such as the potentiation of the nitric oxide pathway, or the inhibition of the RhoA-Rho kinase pathway. The last part of this review deals with drug-induced RP. Among various medications, beta-blockers, interferons, tyrosine-kinase inhibitors or cytotoxic agents such as bleomycin are involved.Thérapie 03/2014; 69(2):115-128. DOI:10.2515/therapie/2013068 · 0.51 Impact Factor