期刊
REVIEWS IN CARDIOVASCULAR MEDICINE
卷 21, 期 2, 页码 163-179出版社
IMR PRESS
DOI: 10.31083/j.rcm.2020.02.597
关键词
Pulmonary hypertension; FDA; clinical trial; treatment; gene therapy; epigenetics
资金
- NIH [R01 HL133554, 5T32HL007824-22]
- AHA [18IPA34170321]
- Cardiovascular Medical Research and Education Fund (CMREF)
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.
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