MICA: Pulmonary Hypertension: intensification and personalisation of combination Rx (PHoenix)

肺动脉高压（PAH）是一种由肺部小血管重构和收缩引起的毁灭性疾病。批准的疗法通过靶向三种不同的生物途径来减少收缩。由于副作用和/或疾病恶化而改变治疗方法。然而，由于需要侵入性的、基于医院的调查来评估疾病的严重程度并证明治疗的益处，因此没有办法将患者与对其疾病有效的药物相匹配。通过MRC对概念的信心资助的可行性研究，我们已经开发了使用监管部门批准的微创监测器进行关键测量的能力，可以在患者在家时评估疾病严重程度和治疗效果。通过监管部门批准的临床使用的在线门户网站，每天将数据传递给护理团队，从而可以对单个患者的治疗效果进行早期远程评估。在肺动脉高压（PAH）患者中，本研究将评估植入式/远程技术的能力，以早期评估临床疗效，并为患者匹配适合他们的药物。40名接受指南推荐治疗的多环芳烃患者将植入经批准的医疗设备，并建立远程监测。在口服前列环素IP受体激动剂和可溶性鸟苷酸环化酶刺激剂的随机交叉研究中，将监测生理、活动和生活质量。该研究将以一种以前无法实现的方式评估治疗强化和降级的生理学。如果成功，该技术方法将促进该领域的新研究设计，为肺动脉高压患者提供一个有可能改变实验医学、治疗开发和个性化药物的平台。

Pulmonary arterial hypertension (PAH) is a devastating disease driven by remodelling and constriction of the small blood vessels of the lungs. Approved therapies reduce constriction by targeting three distinct biological pathways. Therapies are changed due to side effects and/or disease worsening. However, due to the invasive, hospital-based investigations required to assess disease severity and demonstrate benefit of treatment, there is no way to match patients to drugs effective for their disease. Through an MRC Confidence-in-Concepts funded feasibility study we have developed the capacity to use regulatory approved, minimally-invasive monitors to make key measurements that allow assessment of disease severity and treatment effect while patients are at home. Data is relayed daily to care teams through regulatory approved, clinically used online portals, making early, remote evaluation of treatment benefit in an individual patient possible. In patients with pulmonary arterial hypertension (PAH) this study will: evaluate the capacity of implantable/remote technology for early evaluation of clinical efficacy and matching patients to drugs that work for them.Forty patients with PAH established on guideline recommended therapy will be implanted with approved medical devices and remote monitoring established. Physiology, activity and quality-of-life will be monitored during a randomised cross-over study of oral prostacyclin IP receptor agonist and soluble guanylate cyclase stimulator. The study will assess the physiology of therapeutic intensification and de-escalation in a manner previously unachievable. If successful, the technological approach will facilitate novel study designs in the area, providing a platform that has the potential to transform experimental medicine, therapeutic development and personalised medicine for patients with pulmonary hypertension.