Long-term treatments for systemic sclerosis: what are the perspectives?
Mouthon, L.; Agard, C.; Garcia De La Peña-Lefebvre, P.; Guillevin, Lïc.
Annales de Medecine Interne 153(4): 265-275
2002
ISSN/ISBN: 0003-410X PMID: 12218893 Document Number: 543756
Systemic sclerosis (SSc) is responsible for fibrosis of the dermis and other organs as well as vascular abnormalities. While the pathogenesis of SSc is continually being better understood, there is still no single therapeutic agent that has been shown to increase survival in a prospective randomized trial. Traditional medications such as colchicine and D-penicillamine are disappointing in clinical practice, and the latter one failed to clearly show benefit when tested in a prospective placebo controlled trial comparing conventional high dose versus low dose. Conversely, new disease modifying agents are emerging such as cyclophosphamide (CYC) in interstitial pulmonary disease and stem cell autograft after high dose CYC therapy in patients who develop visceral involvement in the three first years of evolution of the disease. Organ specific therapy may show dramatic benefit, such as angiotensin converting enzyme inhibitors in renal crisis and epoprostenol in primary pulmonary hypertension. We will try to review disease modifying agents available in SSc and emphase new therapeutic agents that are currently being evaluated, including vasodilatators, anti-inflammatory, anti-fibrosing agents and immunosuppressive molecules.