Nathan Steven D, Fernandes Peter, Psotka Mitchell, Vitulo Patrizio, Piccari Lucilla, Antoniou Katerina, Nikkho Sylvia M, Stockbridge Norman
Advanced Lung Disease and Transplant Program, Inova Heart and Vascular Institute Falls Church Virginia USA.
Bellerophon Therapeutics Inc., Regulatory, Safety and Quality Department Warren New Jersey USA.
Pulm Circ. 2022 Oct 1;12(4):e12178. doi: 10.1002/pul2.12178. eCollection 2022 Oct.
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.
与间质性肺疾病（ILD）相关的肺动脉高压（PH）是PH药物临床试验的一个有吸引力的目标。要使此类研究取得成功，需要考虑许多因素。最有可能对干预措施产生反应的患者表型需要权衡实质性肺部疾病的程度与血流动力学损害的严重程度。纳入标准不应过于严格，以便能够招募到患者。试验应具有足够的持续时间以达到所选的终点，该终点应反映患者的感受、功能或生存情况。本文总结了先前关于PH-ILD的研究，并提供了一个可供考虑的研究类型框架。还讨论了PH-ILD试验中的纳入标准、临床试验终点和药物警戒。通过从先前研究中吸取的经验教训，也为未来PH-ILD的临床试验提供了建议和指导。
