Pulmonary Hypertension is a silent disease and its diagnosis often occurs when it is already at an advanced stage. Pharmacological treatment of Pulmonary Arterial Hypertension (PAH) can be performed with: calcium channel blockers; phosphodiesterase-5 inhibitors; prostanoids; endothelin-receptor antagonists; and, soluble guanylate cyclase stimulators. The use of Riociguat was approved in Brazil by the National Sanitary Surveillance Agency on October 5, 2015 for use in patients with PAH. The objective was to perform a systematic review (SR) of the efficacy of pharmacological treatment of Pulmonary Arterial Hypertension comparing Riociguat with other available medications or with placebo.

Following the steps described in the PRISMA guideline, a search for randomized controlled clinical trials was conducted, in which Riociguat was used alone or in combination with other therapies, in databases MEDLINE, LILACS, Web of Science, Science Direct, Cochrane Library Wiley and in the gray literature (Google Scholar, Capes Bank of Theses and Clinical Trials). EndNote and Mendeley were used as reference managers. Outcomes analyzed were: death; six-minute walk distance (6MWD); World Health Organization (WHO) functional class (improvement, stabilization or worsening); hemodynamic variables (pulmonary vascular resistance, cardiac index and pulmonary-artery pressure); clinical worsening; hospitalization; and, quality of life.

Four hundred and sixty-seven articles were obtained which reduced to 379 after the duplicated articles were removed. After exclusion by title and abstract by two independent reviewers, forty-seven studies remained. Through the gray literature, six studies were obtained, resulting in fifty-three articles being retrieved for full-text review. Five studies were selected to compose the SR. Compared with placebo, Riociguat showed improvements in 6MWD, pulmonary vascular resistance, WHO functional class and time to clinical worsening. Efficacy was maintained after one year of use. Subgroup analysis was performed comparing of treatment-naive patients and patients on background PAH-targeted therapy.

This work may be used as a management and decision support tool, based on the same methodology of a Health Technology Assessment, and may contribute to quality decisions to be taken in relation to the incorporation of new technology.