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


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Abstract

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.

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Выбор новых "конечных точек" в клинических исследованиях, посвященных оценке эффективности лекарственной терапии у пациентов с легочной артериальной гипертонией. - Аннотация. Легочная артериальная гипертония (ЛАГ) характеризуется неблагоприятным прогнозом; по данным современных национальных регистров, 3-летняя выживаемость пациентов с ЛАГ не превышает 60%. В течение последних 10 лет появи­лись лекарственные средства, способные уменьшить симптомы, улучшить качество жизни и замедлить прогрессирование заболевания. Исторически клинические исследования, посвященные ЛАГ, имели фиксированную и достаточно короткую продолжительность и чаще всего в качестве "первичной конечной точки" использовали такой показатель, как изменение пройденного расстояния в тесте с 6-минутной ходьбой. Для дальнейшей оптимизации терапии ЛАГ требуется оценка эффективности лекарственных средств и стратегий терапии с точки зрения их влияния на морбидность и летальность, т.е. на прогноз заболевания. В настоящее время единственным завершенным и опубликованным клиническим исследованием, в котором в качестве "первичной конечной точки" использован комбинированный показатель морбидности и летальности, является исследование SERAPHIN. В нем продемонстрировано, что терапия новым препаратом мацитентаном приводит к снижению морбидности и летальности у пациентов с ЛАГ.
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About the authors

S N Avdeev

Email: serg_avdeev@list.ru

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