PDF(Pubmed)
Abstract：
BACKGROUND: Pulmonary hypertension (PH) is a leading cause of death in patients with systemic sclerosis (SSc). An important component of SSc patient management is early detection and treatment of PH. Recently the threshold for the diagnosis of PH has been lowered to a mean pulmonary artery pressure (mPAP) threshold of > 20 mmHg on right heart catheterization (RHC). However, it is unknown if PH-specific therapy is beneficial in SSc patients with mildly elevated pressure (SSc-MEP, mPAP 21-24 mmHg).
METHODS: The SEPVADIS trial is a randomized, double-blind, placebo-controlled phase 2 trial of sildenafil in SSc-MEP patients with a target enrollment of 30 patients from two academic sites in the United States. The primary outcome is change in six-minute walk distance after 16 weeks of treatment. Secondary endpoints include change in pulmonary arterial compliance by RHC and right ventricular function by cardiac magnetic resonance imaging at 16 weeks. Echocardiography, serum N-terminal probrain natriuretic peptide, and health-related quality of life is being measured at 16 and 52 weeks.
CONCLUSIONS: The SEPVADIS trial will be the first randomized study of sildenafil in SSc-MEP patients. The results of this trial will be used to inform a phase 3 study to investigate the efficacy of treating patients with mild elevations in mPAP.
BACKGROUND: ClinicalTrials.gov Identifier NCT04797286.
METHODS: The SEPVADIS trial is a randomized, double-blind, placebo-controlled phase 2 trial of sildenafil in SSc-MEP patients with a target enrollment of 30 patients from two academic sites in the United States. The primary outcome is change in six-minute walk distance after 16 weeks of treatment. Secondary endpoints include change in pulmonary arterial compliance by RHC and right ventricular function by cardiac magnetic resonance imaging at 16 weeks. Echocardiography, serum N-terminal probrain natriuretic peptide, and health-related quality of life is being measured at 16 and 52 weeks.
CONCLUSIONS: The SEPVADIS trial will be the first randomized study of sildenafil in SSc-MEP patients. The results of this trial will be used to inform a phase 3 study to investigate the efficacy of treating patients with mild elevations in mPAP.
BACKGROUND: ClinicalTrials.gov Identifier NCT04797286.
摘要：
背景：肺动脉高压（PH）是系统性硬化症（SSc）患者死亡的主要原因。SSc患者管理的重要组成部分是早期发现和治疗PH。最近，在右心导管插入术（RHC）中，诊断PH的阈值已降低至平均肺动脉压（mPAP）阈值>20mmHg。然而,尚不清楚PH特异性治疗是否对压力轻度升高的SSc患者有益（SSc-MEP，mPAP21-24mmHg）。
方法：SEPVADIS试验是一项随机，双盲,西地那非在SSc-MEP患者中的安慰剂对照2期试验,目标纳入来自美国两个学术中心的30名患者.主要结果是治疗16周后6分钟步行距离的变化。次要终点包括16周时RHC引起的肺动脉顺应性变化和心脏磁共振成像引起的右心室功能变化。超声心动图,血清N末端脑钠肽，在16周和52周时测量与健康相关的生活质量。
结论：SEPVADIS试验将是西地那非在SSc-MEP患者中的第一个随机研究。该试验的结果将用于告知3期研究，以研究治疗mPAP轻度升高患者的疗效。
背景：ClinicalTrials.gov标识符NCT04797286。
方法：SEPVADIS试验是一项随机，双盲,西地那非在SSc-MEP患者中的安慰剂对照2期试验,目标纳入来自美国两个学术中心的30名患者.主要结果是治疗16周后6分钟步行距离的变化。次要终点包括16周时RHC引起的肺动脉顺应性变化和心脏磁共振成像引起的右心室功能变化。超声心动图,血清N末端脑钠肽，在16周和52周时测量与健康相关的生活质量。
结论：SEPVADIS试验将是西地那非在SSc-MEP患者中的第一个随机研究。该试验的结果将用于告知3期研究，以研究治疗mPAP轻度升高患者的疗效。
背景：ClinicalTrials.gov标识符NCT04797286。
