Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: a European Scleroderma Trials and Research (EUSTAR) analysis.

Objectives: Mortality and worsening of organ function are desirable endpoints for clinical trials in systemic sclerosis (SSc). The aim of this study was to identify factors that allow enrichment of patients with these endpoints, in a population of patients from the European Scleroderma Trials and Research group database.

Methods: Inclusion criteria were diagnosis of diffuse SSc and follow-up over 12±3 months.

Progressive skin fibrosis is associated with a decline in lung function and worse survival in patients with diffuse cutaneous systemic sclerosis in the European Scleroderma Trials and Research (EUSTAR) cohort.

Objectives: To determine whether progressive skin fibrosis is associated with visceral organ progression and mortality during follow-up in patients with diffuse cutaneous systemic sclerosis (dcSSc).

Methods: We evaluated patients from the European Scleroderma Trials and Research database with dcSSc, baseline modified Rodnan skin score (mRSS) ≥7, valid mRSS at 12±3 months after baseline and ≥1 annual follow-up visit. Progressive skin fibrosis was defined as an increase in mRSS >5 and ≥25% from baseline to 12±3 months.

Riociguat for the treatment of pulmonary hypertension: Chinese subgroup analyses and comparison.

Objective: PATENT-1 and CHEST-1 were pivotal, international phase III trials assessing riociguat for pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). Here we compare Chinese patients from these studies with the overall populations, and report the clinical effect and safety of riociguat in Chinese patients with PAH and CTEPH.

Methods: PATENT-1 was a 12-week, randomised, double-blind, placebo-controlled trial of riociguat (maximum 2.

Use of responder threshold criteria to evaluate the response to treatment in the phase III CHEST-1 study.

Background: In the Chronic Thromboembolic Pulmonary Hypertension Soluble Guanylate Cyclase - Stimulator Trial 1 (CHEST-1) study, riociguat improved 6-minute walking distance (6MWD) vs placebo in patients with inoperable chronic thromboembolic pulmonary hypertension or persistent/recurrent pulmonary hypertension after pulmonary endarterectomy. In this study, the proportion of patients who achieved responder thresholds that correlate with improved outcome in patients with pulmonary arterial hypertension was determined at baseline and at the end of CHEST-1.

Methods: Patients received placebo or riociguat individually adjusted up to 2.

Use of clinically relevant responder threshold criteria to evaluate the response to treatment in the phase III PATENT-1 study.

Background: In PATENT-1, riociguat significantly improved 6-minute walking distance (6MWD) and a range of secondary end-points in patients with pulmonary arterial hypertension (PAH). We investigated whether riociguat increased the proportion of patients achieving clinically relevant responder thresholds compared with placebo during PATENT-1.

Methods: In PATENT-1, a randomized, double-blind study, treatment-naïve patients or patients on background PAH-targeted therapy with symptomatic PAH received 12 weeks of treatment with placebo, riociguat up to 2.

Advancing clinical trial design in pulmonary hypertension.

In pulmonary hypertension, as in many other diseases, there is a need for a smarter approach to evaluating new treatments. The traditional randomized controlled trial has served medical science well, but constrains the development of treatments for rare diseases. A workshop was established to consider alternative clinical trial designs in pulmonary hypertension and here discusses their merits, limitations and challenges to implementation of novel approaches.

The Virtual International Stroke Trials Archive.

Background And Purpose: Stroke has global importance and it causes an increasing amount of human suffering and economic burden, but its management is far from optimal. The unsuccessful outcome of several research programs highlights the need for reliable data on which to plan future clinical trials. The Virtual International Stroke Trials Archive aims to aid the planning of clinical trials by collating and providing access to a rich resource of patient data to perform exploratory analyses.

Vardenafil improves sexual function and treatment satisfaction in couples affected by erectile dysfunction (ED): a randomized, double-blind, placebo-controlled trial in PDE5 inhibitor-naïve men with ED and their partners.

Introduction: Erectile dysfunction (ED) is a common condition with diverse sequelae affecting men and their women partners.

Aim: The FINDER study aimed to evaluate the efficacy and tolerability of the phosphodiesterase type-5 (PDE5) inhibitor, vardenafil, in a broad population of PDE5-naïve men with ED of different etiologies and severity and included their partners' assessments of treatment with vardenafil.

Methods: Men with ED (N = 260) were randomized to receive either vardenafil or placebo in a multicenter, flexible-dose study.