Pulmonary hypertension in interstitial lung disease: Clinical trial design and endpoints: A consensus statement from the Pulmonary Vascular Research Institute's Innovative Drug Development Initiative—Group 3 Pulmonary Hypertension

Abstract

Pulmonary hypertension (PH) associated with interstitial lung disease (ILD) is an attractive target for clinical trials of PH medications. There are many factors that need to be considered to prime such studies for success. The patient phenotype most likely to respond to the intervention requires weighing the extent of the parenchymal lung disease against the severity of the hemodynamic impairment. The inclusion criteria should not be too restrictive, thus enabling recruitment. The trial should be of sufficient duration to meet the chosen endpoint which should reflect how the patient feels, functions, or survives. This paper summarizes prior studies in PH‐ILD and provides a framework of the type of studies to be considered. Inclusion criteria, clinical trial endpoints, and pharmacovigilance in the context of PH‐ILD trials are also addressed. Through lessons learnt from prior studies, suggestions and guidance for future clinical trials in PH‐ILD are also provided.

Cluster analysis identifies novel real-world lung disease-pulmonary hypertension sub-phenotypes: implications for treatment response

“…53 While recent guidelines allow for consideration of PAH therapy in patients with lung disease and PVR > 5 WU, cumulatively, our results and those previously published highlight the importance of prospective studies, particularly within subgroups enriched for treatment response, possibly with multicomponent endpoints. 54,55 Finally, our study highlights the importance of endpoints that capture clinical deterioration including morbidity and mortality and is the first to examine long-term treatment effects within Group 3 PH subgroups. 32,55…”