Researchers from China have traced the remarkable 30-year evolution of pulmonary arterial hypertension treatment, from a disease with no effective options to one on the cusp of a potential cure. What was once described as the “cancer of cardiovascular diseases” — with a median survival of just 2.8 years after diagnosis — has become a condition with multiple effective treatment options and significantly improved outcomes.
The journey began in 1995 with the Food and Drug Administration (FDA) approval of epoprostenol, the first targeted therapy for pulmonary arterial hypertension, which marked a turning point in the field. This was followed by the development of drugs targeting three key pathways — the endothelin pathway, the nitric oxide pathway, and the prostacyclin pathway — expanding treatment options considerably over the following two decades.
Treatment strategies also evolved, shifting from initial monotherapy to early combination therapy targeting multiple pathways simultaneously, which has proven significantly more effective, particularly for high-risk patients.
Alongside advances in treatment, risk assessment tools have become an essential part of clinical practice. Models such as COMPERA and REVEAL allow clinicians to classify patients into low, intermediate, and high-risk categories based on indicators including cardiac function, exercise capacity, and natriuretic peptide levels. The goal of treatment is now explicitly defined as achieving and maintaining a low-risk profile — a concept that has been progressively refined from the 2015 ESC/ERS guidelines through to the four-tier COMPERA 2.0 model introduced in the 2022 guidelines.
Treatment goals have risen in parallel: from simply improving exercise tolerance, to reducing mortality risk, to the most ambitious goal yet — reversing pulmonary vascular remodelling entirely. This last frontier has been brought closer by sotatercept, approved in 2024, which works by directly targeting the core biological mechanisms driving the disease rather than merely dilating blood vessels.
Looking ahead, say the authors, the field is moving towards a “3+1+X” spectrum of targeted therapies, with the ultimate ambition of one day curing pulmonary hypertension altogether.
Read more at this link on The Chinese Medical Journal
Citation
Thirty Years of Targeted Drug Therapy for Pulmonary Hypertension: A Paradigm Shift from “Vasodilator” to “Reversing Remodeling”, Xiong Changming, Zhai Zhenguo, Liu Zhihong, Publication Date: May 19, 2026, DOI: 10.3760/cma.j.cn112137-20260130-00337