PROPULSE-Sch: Long-Term Evaluation of Selexipag in Schistosomiasis-Associated Pulmonary Arterial Hypertension Using a Propensity Score-Matched Mirror Cohort
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Time to Clinical Worsening
研究概览
简要总结
Schistosomiasis-associated pulmonary arterial hypertension is a serious condition that can lead to shortness of breath, heart failure, frequent hospitalizations, and early death. Although treatments for pulmonary arterial hypertension have improved over time, patients with this specific cause of the disease are often not included in long-term studies.
Selexipag is an oral medication used to treat pulmonary arterial hypertension and is part of routine clinical care in Brazil. Its long-term effects in patients with schistosomiasis-associated pulmonary arterial hypertension are not well understood.
The PROPULSE-Sch study aims to evaluate long-term clinical outcomes in patients with schistosomiasis-associated pulmonary arterial hypertension who received selexipag, compared with similar patients who did not receive this medication before it became available at the study center.
This is an observational study using data from routine medical care. All treatments are prescribed by the treating physicians, and participation in the study does not change patient care. The results may help improve understanding of long-term outcomes and support treatment decisions in this population.
详细描述
Schistosomiasis-associated pulmonary arterial hypertension (PAH-Sch) is a prevalent cause of pulmonary arterial hypertension in endemic regions and is associated with significant morbidity and premature mortality. Despite advances in targeted therapies for pulmonary arterial hypertension, patients with PAH-Sch remain underrepresented in long-term studies, particularly in real-world clinical settings.
Selexipag, an oral selective prostacyclin IP receptor agonist, has demonstrated clinical and hemodynamic benefits in pulmonary arterial hypertension. Following its incorporation into routine clinical practice in Brazil, selexipag has been increasingly used in eligible patients with PAH-Sch. However, evidence regarding its long-term outcomes in this specific population is limited.
PROPULSE-Sch is a single-center, observational, longitudinal study with an ambispective design, combining retrospective data and prospective follow-up. The study evaluates long-term clinical outcomes associated with exposure to selexipag in patients with PAH-Sch, compared with a mirror cohort of clinically similar patients who did not receive selexipag prior to its availability at the center. To reduce confounding and indication bias inherent to observational comparisons, analyses are conducted using propensity score matching.
All treatment decisions, including initiation and intensification of therapy, are made exclusively by the treating physicians as part of routine clinical care. The study does not mandate any intervention, treatment assignment, or protocol-driven management. Data are obtained from medical records and standard follow-up visits. By using real-world data and robust observational methods, this study aims to contribute clinically relevant evidence on long-term outcomes in schistosomiasis-associated pulmonary arterial hypertension.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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 associated with schistosomiasis (PAH-Sch).
- •Diagnosis of pre-capillary pulmonary arterial hypertension confirmed by right heart catheterization, performed at any time prior to the index date (T0), as documented in the medical record.
- •Evidence of schistosomiasis infection, including epidemiological history and ultrasonographic findings compatible with hepatosplenic schistosomiasis.
- •Previous antiparasitic treatment for schistosomiasis.
- •Clinical stability at the index date, defined as absence of progressive right heart failure or clinical worsening within the previous 12 weeks.
- •World Health Organization (WHO) functional class I-III at the index date.
- •Stable background pulmonary arterial hypertension-specific therapy with a phosphodiesterase-5 inhibitor and/or endothelin receptor antagonist for at least 12 weeks prior to the index date.
- •For the treated cohort: initiation of oral selexipag as part of routine clinical care.
- •For the mirror cohort: eligibility for therapeutic escalation at the index date without exposure to selexipag.
排除标准
- •World Health Organization (WHO) functional class IV at the index date.
- •Progressive right heart failure or clinical deterioration within the 12 weeks prior to the index date.
- •Documented formal contraindication to selexipag in the medical record.
- •Insufficient baseline data at the index date to allow clinical characterization or inclusion in propensity score analyses.
研究组 & 干预措施
Selexipag-Treated PAH-Sch Cohort
Adult patients with schistosomiasis-associated pulmonary arterial hypertension who initiated oral selexipag as part of routine clinical care. The index date (T0) is defined as the date of selexipag initiation recorded in the medical record. Patients are followed longitudinally to assess long-term clinical outcomes.
Mirror PAH-Sch Cohort Without Selexipag
Adult patients with schistosomiasis-associated pulmonary arterial hypertension who did not receive selexipag and were followed at the same center prior to its availability. The index date (T0 mirror) is defined as the first visit at which patients met clinical eligibility criteria comparable to those of the treated cohort.
结局指标
主要结局
Time to Clinical Worsening
时间窗: From index date (T0) up to 36 months of follow-up
Time from the index date (T0) to the first occurrence of clinical worsening, defined as any of the following events: all-cause mortality; lung transplantation; non-elective hospitalization due to pulmonary arterial hypertension or right heart failure; therapeutic escalation defined as initiation of parenteral prostacyclin or addition of a new class of pulmonary arterial hypertension-specific therapy; or sustained worsening of World Health Organization functional class confirmed in two consecutive assessments at least 12 weeks apart and accompanied by objective evidence of disease progression, including a ≥15% decrease in six-minute walk distance and/or a ≥30% increase in BNP or NT-proBNP compared with baseline.
次要结局
- Clinical Improvement Composite Outcome(6, 12, 24, and 36 months after index date)
- Change in WHO Functional Class(Up to 36 months of follow-up)
- Change in 6-Minute Walk Distance (6MWD)(Up to 36 months)
- Change in BNP or NT-proBNP Levels(Up to 36 months)
- Hemodynamic Parameters(Up to 36 months of follow-up)
- Risk Stratification Scores(Up to 36 months of follow-up)
- Treatment Tolerability(Up to 36 months of follow-up)
- Hemodynamic Parameters(Up to 36 months)
研究者
Caio Júlio César dos Santos Fernandes
Principal investigator
University of Sao Paulo General Hospital