Idiopathic- and scleroderma-related pulmonary arterial hypertension: outcomes and QOL on bosentan

Idiopathic pulmonary arterial hypertension (previously known as primary pulmonary hypertension) is a devastating disease of insidious onset, late diagnosis, progressive functional disability and poor prognosis. However, treatment with pulmonary arterial hypertension-specific agents has already changed the outlook wherever these agents are accessible. The process of economic evaluation of treatment for idiopathic pulmonary arterial hypertension is challenging due to limitations common to clinical studies in rare diseases, such as small patient numbers limiting survival information, lack of directly comparable trials for the efficacy of different drugs and a paucity of quality of life measurements. Bosentan (Tracleer), Actelion), has proven effective in clinical trials, and is the most frequently used pulmonary arterial hypertension-specific agent worldwide. It has additionally undergone a comprehensive quality of life trial and economic evaluation as therapy for idiopathic pulmonary arterial hypertension and pulmonary arterial hypertension associated with scleroderma. This review explores the role of bosentan in providing cost-effective therapy compared with alternative treatments, and addresses the future considerations required to ensure accessible patient care for idiopathic pulmonary arterial hypertension and pulmonary arterial hypertension associated with scleroderma.