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.
SourceAvailable from: Matthieu Roustit[Show abstract] [Hide abstract]
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 · 0.40 Impact Factor
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ABSTRACT: The aim of the study was to examine the effect of bosentan on blood flow in the hand in a subset of patients who had reduced blood flow relative to healthy subjects. Additionally, the relationship between blood flow in the hands of SSc patients and the presence of digital ulcers (DUs) was assessed.Rheumatology (Oxford, England) 08/2014; DOI:10.1093/rheumatology/keu300 · 4.44 Impact Factor
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ABSTRACT: Introduction: The treatment of systemic sclerosis (SSc) still represents a challenge for all physicians because of the complexity of this disease. However, new insights into pathophysiology are driving new approaches to its treatment by acting on key targets of pathogenesis. Areas covered: The management of SSc consists of a symptomatic therapy and a disease modifying therapy. The aim of symptomatic treatment is to control different symptoms, clinical manifestations and organ-based complications while disease modifying therapy interferes with the main pathogenetic mechanisms in order to obtain remission, to stop disease progression and if possible to reverse disease-related skin and internal organ changes. This article will provide an overview of the different treatments for the most common features of SSc. A Medline search was focused on most relevant treatment literature contribution published in the past decade. Expert opinion: As for symptomatic treatment, calcium channel blockers (CCBs) should be considered as the first-line therapy for Raynaud's phenomenon (RP). In case of intolerance or ineffectiveness, a phosphodiesterase type 5 (PDE5) inhibitor alone or in association with CCB can be started. As for pulmonary arterial hypertension (PAH), both bosentan, ambrisentan and sildenafil, can be used as first-line treatments. In patients with organ involvement at risk for progression, cyclophosphamide (CYC) should be considered the disease modifying agent of choice. In case of contraindications to CYC, rituximab (RTX) and mycophenolate mofetil (MMF) have to be taken into account. After induction treatment, maintenance with MMF or azathioprine (AZA) has to be started.