Current FDA-approved therapies for pulmonary arterial hypertension improve quality of life but have limited effects on survival, with median survival remaining less than three years from diagnosis.
This review highlights that while current PAH therapies improve symptoms and hemodynamics, they do not significantly alter survival, underscoring the need for emerging treatments like stem cell and gene therapies.
Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease of multifactorial etiology. Most of the available drugs and FDA-approved therapies for treating pulmonary hypertension attempt to overcome the imbalance between vasoactive and vasodilator mediators, and restore the endothelial cell function. Traditional medications for treating PAH include the prostacyclin analogs and receptor agonists, phosphodiesterase 5 inhibitors, endothelin-receptor antagonists, and cGMP activators. While the current FDA-approved drugs showed improvements in quality of life and hemodynamic parameters, they have shown only very limited beneficial effects on survival and disease progression. None of them offers a cure against PAH, and the median survival rate remains less than three years from diagnosis. Extensive research efforts have led to the emergence of innovative therapeutic approaches in the area of PAH. In this review, we provide an overview of the current FDA-approved therapies in PAH and discuss the associated clinical trials and reported-side effects. As recent studies have led to the emergence of innovative therapeutic approaches in the area of PAH, we also focus on the latest promising therapies in preclinical studies such as stem cell-based therapies, gene transfer, and epigenetic therapies.
Bisserier et al. (Tue,) conducted a review in Pulmonary arterial hypertension. FDA-approved therapies and emerging treatments was evaluated. Current FDA-approved therapies for pulmonary arterial hypertension improve quality of life but have limited effects on survival, with median survival remaining less than three years from diagnosis.