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Recent advances in the treatment of idiopathic PAH have resulted in increased survival
Idiopathic pulmonary arterial hypertension (PAH), formerly known as primary pulmonary hypertension, is characterised by raised pulmonary artery pressure and pulmonary vascular resistance in the absence of underlying significant cardiopulmonary or other medical disease. Idiopathic PAH is a progressive disorder that usually culminates in right ventricular failure and death. Moreover, patients with idiopathic PAH are often severely limited on exertion by dyspnoea and fatigue, and thus suffer from a poor quality of life.
Despite clinical recognition of idiopathic PAH for more than 100 years, there have been few effective therapeutic options until recently. Given the previous absence of effective pharmacological therapy, surgical removal of the disease by lung transplantation has been an important therapeutic option for patients with idiopathic PAH. Lung transplantation is associated with rapid and sustained improvement in pulmonary haemodynamics.1 Moreover, in individual patients with idiopathic PAH who do not respond to medical treatment, timely lung transplantation will often be associated with improved survival even though long term survival after lung transplantation remains poor.2,3
One of the first long term pharmacological approaches used for treating idiopathic PAH was systemic anticoagulation. In patients with idiopathic PAH, pulmonary microvascular thrombosis is present histologically and there is laboratory evidence of a procoagulant haematological state.4,5 Moreover, both retrospective and non-randomised cohort studies have suggested a survival benefit of systemic anticoagulation in idiopathic PAH.6,7 Although not formally studied in a randomised controlled trial, systemic anticoagulation is recommended for the majority of patients with PAH in the absence of a clear contraindication based on assessment of individual bleeding risk.8
SURVIVAL BENEFIT OF NEW TREATMENTS IN IDIOPATHIC PAH
Ongoing basic biological and clinical research has led to tremendous advances in our understanding of the pathobiology of idiopathic PAH. Moreover, an increasing number of …