Abstract
Systemic sclerosis (SSc) is a multi-system disease characterized by skin fibrosis and visceral disease. Therapy is organ and pathogenesis targeted. In this review, we describe novel strategies in the treatment of SSc. Utilizing the MEDLINE and the COCHRANE REGISTRY, we identified open trials, controlled trials, for treatment of SSc from 1999 to April 2005. We used the terms scleroderma, systemic sclerosis, Raynaud's phenomenon, pulmonary hypertension, methotrexate, cyclosporin, tacrolimus, relaxin, low-dose penicillamine, IVIg, calcium channel blockers, losartan, prazocin, iloprost, N-acetylcysteine, bosentan, cyclophosphamide, lung transplantation, ACE inhibitors, anti-thymocyte globulin, and stem cell transplantation. Anecdotal reports were omitted. Methotrexate, cyclosporin, tacrolimus, relaxin, low-dose penicillamine, and IVIg may be beneficial in improving the skin tightness in SSc. Calcium channel blockers, the angiotensin II receptor type 1 antagonist losartan, prazocin, the prostacyclin analogue iloprost, N-acetylcysteine and the dual endothelin-receptor antagonist bosentan may be beneficial for Raynaud's phenomenon. Epoprostenol and bosentan are approved for therapy of pulmonary hypertension (PAH). Other options under investigation include intravenous or aerolized iloprost. Cyclophosphamide (CYC) pulse therapy is effective in suppressing active alveolitis. Stem cell and lung transplantation is a viable option for carefully selected patients. Renal crisis can be effectively managed when hypertension is aggressively controlled with angiotensin converting enzyme (ACE) inhibitors. Patients should continue taking ACE inhibitors even after beginning dialysis in hope of discontinuing dialysis. Anti-thymocyte globulin and mycophenolate mofetil appear safe in SSc. The improvement in skin score and the apparent stability of systemic disease during the treatment period suggest that controlled studies of these agents are justified. Stem cell transplantation is under investigation for severe disease. Novel therapies are currently being tested in the treatment of SSc and have the potential of modifying the disease process and overall clinical outcome. The evaluation of these studies is still a difficult process.
Highlights
Systemic sclerosis (SSc) is a multi-system disease characterized by skin fibrosis and visceral disease
With SSc and DLCO of less than 95%, received beraprost sodium for at least 12 months
Among patients with primary pulmonary artery hypertension (PAH) or PAH related to SSc, randomized, double blind, placebo-controlled studies have demonstrated that bosentan therapy (62.5 – 250 mg twice daily) improves hemodynamics, exercise capacity and functional class (Channick et al 2001, Rubin et al 2002)
Summary
Systemic sclerosis (SSc) is a multi-system disease characterized by skin fibrosis and visceral disease. The pathophysiology includes vascular damage, fibroblast proliferation, collagen production and activation of the immune system. Clinical manifestations include thickening of the skin, Raynaud’s phenomenon, pulmonary artery hypertension (PAH) and pulmonary fibrosis, renal disease and involvement of other visceral organs. There is no single medication for the constellation of manifestations, rather therapy is organ and pathogenesis targeted (Table I). We describe novel strategies in the treatment of SSc
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
