Conference: 2014 International PHA Conference and Scientific Sessions
Release Date: 06.22.2014
Presentation Type: Abstracts
File Download: 2014 Conference Abstract - Badesch D
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Background: In the Phase III PATENT-1 study, riociguat, a novel soluble guanylate cyclase stimulator, was well tolerated and significantly improved 6-minute walking distance as well as consistently improving a range of clinically relevant secondary endpoints in patients with pulmonary arterial hypertension (PAH). The objective of this analysis was to evaluate the impact of riociguat on health-related quality of life (HRQoL) during PATENT-1.
Methods: PATENT-1 was a 12-week, double-blind, randomized, placebo-controlled study. The PAH-disease-specific (Living with Pulmonary Hypertension [LPH]) questionnaire that has been validated for PAH, and the generic EuroQol 5-Dimension (EQ-5D) questionnaire were completed by patients at baseline and at Week 12. The change from baseline to Week 12 for the total population, key subgroups (treatment-naïve and pre-treated patients), and sub-scores were examined. Relationships between HRQoL and other clinical endpoints were evaluated.
Results: A total of 254 patients received riociguat (individual dose adjustment up to 2.5 mg three times daily) and 126 patients received placebo. At Week 12, there was a trend towards a higher EQ-5D score for patients treated with riociguat compared with those patients treated with placebo (least-squares [LS] mean difference +0.06 [95% CI: 0.01 to 0.11]; p=0.066). Improvements from baseline in EQ-5D scores at Week 12 were observed in the pre-treated patient subgroup (LS mean difference +0.08 [95% CI: 0.004 to 0.15]) and to a lesser extent in the treatment-naïve patient subgroup (LS mean difference +0.05 [95% CI: –0.03 to 0.12]). A significant improvement from baseline in the LPH total score was seen at Week 12 in riociguat-treated patients compared with placebo-treated patients (LS mean difference –6.2 [95% CI: –9.8 to –2.5]; p=0.0019). Both treatment-naïve and pre-treated patient subgroups demonstrated similar results for the LPH total score, improving from baseline by –7.2 (95% CI: –12.3 to –2.2) and –5.1 (95% CI: –10.3 to 0.2) at Week 12, respectively. In addition, the LPH physical score improved in patients treated with riociguat compared with no change in placebo-treated patients (LS mean difference –3.3 [95% CI: –4.9 to –1.7]; p<0.0001).
Conclusions: Patients with PAH treated with riociguat reported improvements in both disease-specific and generic measures of HRQoL at Week 12 compared with baseline.TYPE: Clinical Science