Pulmonary Hypertension Knowledge Sharing Platform ’s Post

September brought significant advances across treatment, diagnostics, and patient engagement in pulmonary hypertension and lung transplantation. Major Clinical Trials and Treatment Advances 🔹 Results of the HYPERION study were presented at ERS 2025 and simultaneously published in the New England Journal of Medicine, showing that sotatercept dramatically reduces clinical worsening in newly diagnosed PAH patients. 🔹 In contrast, a large French registry analysis of over 1,500 PAH patients challenged conventional practice by finding no survival benefit from anticoagulation therapy. 🔹 Several pharmaceutical developments advanced the treatment pipeline: Gossamer Bio entered an option agreement with Respira Therapeutics, Inc. Therapeutics to develop RT234 for PAH and PH-ILD, complementing their existing inhaled candidate seralutinib. AllRock Bio raised $50 million to advance ROC-101 into Phase 2a trials for PAH and PH-ILD. United Therapeutics Corporation Therapeutics announced that their TETON-2 pivotal study of Tyvaso met its primary endpoint for idiopathic pulmonary fibrosis treatment, with data on their investigational treprostinil transdermal patch system presented at the US Pulmonary Hypertension Association symposium. Diagnostic and Mechanistic Insights 🔹 Artificial intelligence made inroads into PAH diagnosis, with research demonstrating that deep learning models can identify congenital heart disease-associated PAH on routine chest X-ray, and a Franco-German study revealed new insights into how BMP-9 regulates lung vessels, opening doors for future therapeutic approaches. 🔹 In lung transplantation, researchers identified alveolar septal width as a histological predictor of chronic lung allograft dysfunction and survival, providing clinicians with a new prognostic tool. Meanwhile, a review in Transplantation Direct explored the emerging role of extracorporeal photopheresis in transplantation and other conditions. Risk Assessment and Clinical Guidelines 🔹 The ISHLT - International Society for Heart and Lung Transplantation for published an important consensus statement on understanding risk assessment in PAH, providing standardized approaches for clinicians to stratify patients and guide treatment decisions. Patient Engagement and Advocacy 🔹 Patient empowerment took center stage with multiple initiatives including a symposium at the ERS Annual Congress in Amsterdam where patient advocates Louise Bouman - van der Waal and Gergely Meszaros JD, MSc made the case for the inclusion of the patient perspective in PAH risk assessment evaluation. 🔹 The Pulmonary Vascular Research Institute (PVRI)'s Patient Engagement and Empowerment Workstream released an 11-part series titled "How to empower yourself & others" to support PAH patients. 🔹 The PHAWARE GLOBAL ASSOCIATION podcast launched a special series on "The Rising Crisis of Meth-Associated Pulmonary Hypertension in America," addressing an often-overlooked patient population.

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