Authors' recommendations: Pulmonary arterial hypertension (PAH) refers to the vascular remodeling of the small pulmonary arteries that is associated with elevated pulmonary arterial pressure (PAP) and eventually right ventricular failure (RVF). Symptoms of PAH begin as shortness of breath at exertion, later progressing to dyspnea with normal activities, and ultimately dyspnea at rest. Patients with advanced PAH are unable to perform any activity without shortness of breath or chest pain. PAH is an inexorable, progressive, and lethal disease. Incidence estimates vary and suggest a range of 5 to 52 cases per 1 million persons. Patients who remain untreated have an estimated survival rate of 2.8 years with 1-, 3-, and 5-year survival rates of 68%, 48%, and 34%, respectively. Current therapy may yield average survival after diagnosis of 5 to 7 years in adults. Description of Technology: This health technology assessment focuses on the use of treprostinil sodium (Remodulin) as a continuous subcutaneous (SC) infusion for adults with World Health Organization (WHO) Group 1 PAH with WHO or New York Heart Association (NYHA) functional classes II to IV (mild to severe symptoms). Treprostinil is a tricyclic benzindene analog of prostacyclin with similar antiplatelet and vasodilatory actions, including acute pulmonary vasodilation. Treprostinil exhibits an absolute bioavailability of 100% after SC infusion. Continuous SC administration of treprostinil achieves steady-state plasma concentrations after approximately 10 hours when given at rates from 1.25 nanograms per kilogram per minute (ng/kg/min) to 22 ng/kg/min. The infusion rate is initiated at 1.25 ng/kg/min. If this initial dose cannot be tolerated because of systemic effects, it is recommended to reduce the infusion rate to 0.625 ng/kg/min. Patient Population: Treprostinil sodium (Remodulin) is indicated in patients with PAH categorized as WHO Group 1 with WHO or NYHA functional class II to IV symptoms and etiologies of idiopathic or heritable PAH, PAH associated with congenital systemic-to-pulmonary shunts, or PAH associated with connective tissue diseases. Treprostinil is indicated for symptom reduction. Clinical Alternatives: Alternatives to treprostinil include prostacyclin derivatives (epoprosteno, oral or inhalant treprostinil, and iloprost), endothelin receptor antagonists (bosentan and ambrisentan), phosphodiesterase-5 inhibitors (sildenafil and tadalafil), and nitric oxide. Patients with PAH who fail drug therapies may undergo surgical procedures including lung transplantation or an atrial septostomy.

Project Status: Completed

Year Published: 2017

URL for published report: The report may be purchased from: http://www.hayesinc.com/hayes/crd/?crd=58886

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: United States

Organisation Name: HAYES, Inc.

Contact Address: 157 S. Broad Street, Suite 200, Lansdale, PA 19446, USA. Tel: 215 855 0615; Fax: 215 855 5218

Contact Name: saleinfo@hayesinc.com

Contact Email: saleinfo@hayesinc.com

Copyright: Winifred S. Hayes, Inc