Key result
A review of 24 phase II-III randomized controlled trials identified novel therapies targeting emerging pathways such as BMPR2 signaling and kinase inhibitors for pulmonary arterial hypertension.
Why the study?
Pulmonary arterial hypertension leads to right-heart failure and fatal outcomes if untreated, warranting an overview of novel pipeline therapies currently in phase II-III randomized controlled trials.
This systematic review highlights novel pipeline treatments for pulmonary arterial hypertension currently in phase II-III trials, emphasizing the need for larger trials to validate safety and efficacy.
Answers are grounded in this paper and cite their sources.
Alerts clinicians to novel PAH pipeline agents; leaves open practice change pending larger confirmatory trials.
Pulmonary arterial hypertension is characterized by elevated blood pressure and pathological changes in the pulmonary arterioles, leading to the development of right-heart failure and potentially fatal outcomes if left untreated. This review aims to provide an overview of novel drugs or formulations and new drug indications for pulmonary arterial hypertension that are currently in phases II-III of randomized controlled trials, and describe the rationale for the use of these targeted therapies, as well as their efficacy, safety profile, and impact on quality of life and survival. The literature research was conducted using data from ClinicalTrials.gov for the period between 1 January 2016 up to 31 December 2022. The population of interest includes individuals aged ≥ 18 years who have been diagnosed with pulmonary arterial hypertension. The review selection criteria included trials with recruiting, enrolling by invitation, active, terminated or completed status in 2022 and 2023. A total of 24 studies were selected for evaluation based on the inclusion and exclusion criteria. This review summarizes the updated information from randomized clinical trials involving novel therapies for pulmonary arterial hypertension. However, larger clinical trials are required to validate their clinical safety and effects. In the future, clinicians should choose therapies based on the patient's individual situation and requirements when developing treatment strategies.
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Novara et al. (2024) conducted a review in Pulmonary Arterial Hypertension. Novel pipeline treatments was evaluated. A review of 24 phase II-III randomized controlled trials identified novel therapies targeting emerging pathways such as BMPR2 signaling and kinase inhibitors for pulmonary arterial hypertension.
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