Department of Medicine, Division of Respirology, University of Calgary, Calgary, Canada.
Published: May 2022
AI Article Synopsis
Article Abstract
Background: Initial combination therapy with an endothelin receptor antagonist (ERA) and riociguat in pulmonary arterial hypertension (PAH) has limited supporting data.
Methods: We performed a prospective, single-arm, open-label trial of riociguat, and ambrisentan for incident PAH patients in functional class III. The primary endpoint was pulmonary vascular resistance (PVR) at 4-months.
Results: Twenty patients (59 ± 13 years old, 85% female) enrolled and 1 died before their 4-month follow-up. Fifteen patients completed a 4-month and 13 completed the 12-month follow-up. At 4-months PVR decreased 54% with an absolute change of -5.8 Wood units (95% CI -4.0; -7.5, p < 0.001). Other hemodynamic variables and risk scores also improved. Six patients discontinued riociguat and 8 discontinued ambrisentan, with 5 (25%) discontinuing both.
Conclusions: These results do not support the routine use of riociguat plus ambrisentan in initial regimens. Future studies are needed to compare this strategy with phosphodiesterase-5 inhibitors and an ERA with respect to tolerability and long-term outcomes.
Pulmonary arterial hypertension (PAH) is a severe lung disease marked by elevated pulmonary arterial pressure, causing symptoms that often lead to late diagnosis and a poor prognosis, with a median life expectancy of only three years.
The condition involves a combination of factors leading to blockage and stiffening of blood vessels in the lungs, resulting in right heart strain and potential failure.
A systematic review of recent studies evaluated various PAH treatments, highlighting that while many drugs show promise, most do not offer a cure and some, like imatinib, have undesirable side effects.
The evidence for the treatment of connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) mostly depends on subgroup or post hoc analysis of randomized controlled trials (RCTs). Thus, we performed a meta-analysis of RCTs that reported outcomes for CTD-PAH. PubMed and EMBASE were searched for CTD-PAH treatment.
* PAH-CTD is progressive and has a poor prognosis, highlighting the importance of early diagnosis and specific treatments available, such as various drug classes including prostacyclin derivatives and endothelin receptor antagonists.
* The manuscript aims to review the latest advancements in treatment strategies, the mechanisms of PAH-targeted drugs, and ongoing research to improve outcomes for patients with PAH-CTD.
: Unilateral agenesis of pulmonary arteries (UAPA) is a rare disease, with approximately 400 cases reported to date. UAPA is often associated with congenital heart disease, and the uncomplicated form is isolated UAPA, which accounts for approximately 30% of all cases of UAPA. The incidence of pulmonary hypertension due to UAPA has been reported to range from 19 to 44%.
