Riociguat improves health‐related quality of life for patients with pulmonary arterial hypertension: results from the phase 4 MOTION study

Abstract

Pulmonary arterial hypertension (PAH) is characterized by progressive dyspnea and exercise limitation and is associated with reduced health-related quality of life. Few clinical studies have evaluated the primary effects of treatment of PAH from the patient perspective. Here, we present the impact of riociguat on patient-reported outcomes (PROs) in treatment-naïve patients with PAH. MOTION (NCT02191137) was an open-label, phase 4 trial of riociguat monotherapy in treatment-naïve patients with PAH. The primary endpoint was the change in total score from baseline to Week 24 in the Living with Pulmonary Hypertension (LPH) questionnaire. The Short Form-12 Health Survey and Work Limitations Questionnaire 8 were also utilized to assess PROs. Other secondary endpoints included change from baseline in World Health Organization functional class (WHO FC), 6-min walk distance (6MWD), Modified Borg Dyspnea Scale, and safety. At week 24 (n ¼ 66), the mean (standard deviation [SD]) total LPH score was 37.17 (24.61), for a mean (SD) change from baseline of À10.99 (22.51). At last visit, with week 24 imputed, the mean (SD) total score was 40.63 (28.38), for a mean (SD) change from baseline of À5.40 (27.8) (n ¼ 75; P ¼ 0.0484). Improvement in LPH questionnaire total score was observed by week 4 and was maintained through week 24. Improvements were observed in WHO FC, Modified Borg Dyspnea Scale, and accelerometer-measured 6MWD at week 24. Treatment with riociguat had a positive impact on PROs in treatment-naïve patients with PAH and was well tolerated, with a similar safety profile to that observed in placebo-controlled phase 3 trials.

A focus on riociguat in the treatment of pulmonary arterial hypertension

“…A phase IV, prospective, single‐arm, open‐label study to measure outcome in patients with PAH not on active treatment ( MOTION ) …”

Riociguat: Clinical research and evolving role in therapy

“…The MOTION study (NCT02191137) was a prospective, multicentre, single‐arm, open‐label, phase IV study to assess the impact of riociguat on patient‐reported outcomes in patients with PAH 94 . The primary endpoint was change from baseline to Week 24 in the LPH questionnaire, a 21‐item, validated, disease‐specific health‐related quality of life assessment tool comprising physical (score 0–40) and emotional (score 0–25) domains; a reduction in score indicates an improvement in health‐related quality of life 95 .…”

“…The MOTION study (NCT02191137) was a prospective, multicentre, single‐arm, open‐label, phase IV study to assess the impact of riociguat on patient‐reported outcomes in patients with PAH. 94 The primary endpoint was change from baseline to Week 24 in the LPH questionnaire, a 21‐item, validated, disease‐specific health‐related quality of life assessment tool comprising physical (score 0–40) and emotional (score 0–25) domains; a reduction in score indicates an improvement in health‐related quality of life. 95 The primary endpoint was met, with a mean ± SD change in LPH score from baseline to Week 24 of −5.4 ± 27.8 ( n = 75, P = .048; the minimally important difference that indicates a clinically meaningful improvement was estimated to be a change from baseline of 7).…”

The achievements of the modern specific therapy of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension: focus on the stimulator of soluble guanylate cyclase riociguat

“…Несмотря на то, что в данном post-hoc-анализе отмечался положительный результат, следует помнить, что риоцигуат не зарегистрирован по показанию ЛАГ-ВПС. Для уверенности в эффективности и безопасности риоцигуата у данной подгруппы пациентов необходимы дальнейшие исследования [12,16] [17].…”