REAPPRAISED: Riociguat in Triple Therapy for Pulmonary Arterial Hypertension - A Single-Center Retrospective Study With Composite Endpoint Evaluation
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 15
- 主要终点
- Composite Clinical Outcome at 6 Months
研究概览
简要总结
Pulmonary arterial hypertension is a rare and serious disease that can lead to heart failure and early death despite modern treatments. Many patients are treated with a combination of medications targeting different disease pathways, but some continue to have an inadequate response.
Riociguat is a medication that acts on the nitric oxide pathway and may be beneficial in patients who do not respond well to phosphodiesterase-5 inhibitors. In routine clinical practice, some patients receiving triple therapy switch from a phosphodiesterase-5 inhibitor to riociguat.
The REAPPRAISED study evaluates outcomes in patients with pulmonary arterial hypertension who switched to riociguat while receiving triple therapy. This observational study uses data from routine clinical care and does not involve any experimental treatment. The results may help improve understanding of the effectiveness and safety of this treatment strategy in real-world practice.
详细描述
Pulmonary arterial hypertension (PAH) is a progressive disease associated with high morbidity and mortality despite the use of combination therapy. Triple therapy including an endothelin receptor antagonist, a prostacyclin pathway agent, and a phosphodiesterase-5 inhibitor is commonly used; however, a subset of patients shows an inadequate clinical response.
Riociguat is a soluble guanylate cyclase stimulator that increases cyclic GMP through both nitric oxide-dependent and nitric oxide-independent mechanisms. Switching from a phosphodiesterase-5 inhibitor to riociguat has shown clinical benefit in selected patients, but data in real-world settings and in patients already receiving triple therapy remain limited.
REAPPRAISED is a single-center, retrospective, observational study conducted in a pulmonary hypertension referral center. The study evaluates clinical outcomes following the switch from a phosphodiesterase-5 inhibitor to riociguat in patients with PAH receiving triple therapy. All treatment decisions were made by the treating physicians as part of routine clinical care. No study-mandated interventions or treatment assignments were performed, and data were obtained from medical records.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults aged 18 years or older.
- •Confirmed diagnosis of pulmonary arterial hypertension (Group 1).
- •Treatment with triple therapy including an endothelin receptor antagonist, a prostacyclin pathway agent, and a phosphodiesterase-5 inhibitor for at least 12 weeks.
- •Switch from a phosphodiesterase-5 inhibitor to riociguat as part of routine clinical care.
- •Availability of minimum data to assess the primary outcome at 6 months.
排除标准
- •Concomitant use of phosphodiesterase-5 inhibitors and riociguat.
- •Inability to confirm vital status or hospitalization outcomes within 6 months.
- •Participation in an interventional clinical trial that mandated the treatment switch.
研究组 & 干预措施
Riociguat Triple Therapy Cohort
Adult patients with pulmonary arterial hypertension receiving triple therapy who switched from a phosphodiesterase-5 inhibitor to riociguat as part of routine clinical care. The index date (T0) is defined as the date of riociguat initiation.
结局指标
主要结局
Composite Clinical Outcome at 6 Months
时间窗: 6 months (24±8 weeks) after switch
Composite clinical outcome assessed at 6 months (24±8 weeks) after the switch to riociguat, defined as achievement of at least two of the following criteria: improvement in six-minute walk distance of at least 10% or 30 meters; World Health Organization functional class I or II; and reduction in BNP levels of at least 30%, in the absence of clinical worsening. Clinical worsening is defined as all-cause mortality, hospitalization due to pulmonary arterial hypertension, or unplanned therapeutic escalation.
次要结局
- Persistence and Time to Discontinuation of Riociguat(Up to 6 months)
- Safety Outcomes(Up to 6 months)
- Health Care Utilization(6 months before and 6 months after switch)
- Functional Changes(From baseline to 6 months)
- Risk Stratification (COMPERA 2.0)(From baseline to 6 months)
研究者
Caio Júlio César dos Santos Fernandes
Principal investigator
University of Sao Paulo General Hospital