Choice of novel endpoints in clinical trials evaluating the efficiency of drug therapy in patients with pulmonary hypertension

• Published in: Terapevtic̆eskii arhiv Vol. 86; no. 3; pp. 88 - 93
• Main Author: Avdeev, S N
• Format: Journal Article
• Language: Russian
• Published: Russia (Federation) "Consilium Medicum" Publishing house 01-01-2014
• Subjects: Clinical Trials as Topic; Endothelin Receptor Antagonists; Endpoint Determination; endpoints; Exercise Test; Humans; Hypertension, Pulmonary - diagnosis; Hypertension, Pulmonary - drug therapy; Hypertension, Pulmonary - mortality; Hypertension, Pulmonary - physiopathology; macitentan; morbidity; mortality; Phosphodiesterase 5 Inhibitors - pharmacology; Prognosis; pulmonary hypertension; Quality of Life; study design; Survival Rate; Treatment Outcome
• ISSN: 0040-3660; 2309-5342

Pulmonary hypertension (PH) is characterized by a poor prognosis: the three-year survival rate in patients with PH is not greater than 60% as evidenced by current national registries. In the past decade, there have been drugs that are able to relieve symptoms of the disease, to slow down its progression, and improve quality of life. Historically, clinical trials dealing with PH have had a fixed and rather short-tern period and most commonly used the index "the change in the distance covered in the 6-minute walk test" as a primary endpoint. Further optimization of PH therapy requires that the efficacy of drugs and the strategies of therapy be evaluated with respect to their effects on morbidity and mortality, i.e. on prognosis. The SERAPHIN trial is now the only completed and published clinical trial using the combined index of morbidity and mortality as a primary endpoint. It demonstrates that that therapy with the novel drug macitentan causes a reduction in morbidity and mortality in patients with PH.