Emerging treatments for pulmonary arterial hypertension

Pulmonary arterial hypertension (PAH) is a rare, progressive disease for which no cure exists. However, improved understanding of underlying pathophysiological mechanisms has led to the development of several effective treatments that improve haemodynamics and functional status.

An overview of emerging pharmacological approaches to the management of PAH is presented.

A Medline search was performed for studies describing novel treatments and potential therapeutic targets relevant to PAH.

Several different treatments that modulate abnormalities in the prostacyclin, endothelin and nitric oxide pathways have shown efficacy in randomised, controlled studies and are now licensed for use for PAH patients with advanced disease. Furthermore, there is now encouraging long-term survival data associated with use of these agents. A number of other targets with therapeutic potential have also been identified, such as serotonin, platelet-derived growth factor and vasoactive intestinal peptide. Recently, strategies involving combinations of different PAH-specific agents have emerged as a promising approach for those failing monotherapy.

The therapeutic options available for PAH has improved considerably in recent years and is likely to expand in the future.