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‘PHoenix’ is an innovative new trial testing how well PAH drugs can be personalised for patients – and it’s set to roll out across more of the UK’s specialist centres soon. We spoke to Dr Frankie Varian of the Sheffield Pulmonary Vascular Disease Unit (pictured) to find out more about the study’s aims, and how it’s been shaped by patients from the start…

The PHoenix trial, which opened for recruitment in June 2023, is a unique study using remote monitoring devices, placed into the lungs and over the heart, to help choose the best medications for people with pulmonary arterial hypertension (PAH).

It’s all about tailoring treatments to the individual by finding out which drugs someone responds best to, using advanced technology to monitor people from their own homes – without the need for them to keep travelling into hospitals.

The study aims to discover whether patients on  ‘dual oral therapy’ (two kinds of tablets) can enjoy a better quality of life by adding one of two additional, already approved, drugs - riociguat or selexipag - to their daily routine.

To help guide treatment for patients from their own home and to help balance the benefit of therapies against side effects, the study uses cutting-edge, remote monitoring technology that provides daily data similar to that gained from a right heart catheterisation. This data is provided wirelessly to the hospital team via mobile phone. 

One tiny device sits in the pulmonary artery and the other just under the skin on the chest. This technology monitors the pulmonary artery pressures and heart rate rhythm activity by sending readings directly to the clinical team and alerting them of any problems.

Patients involved in the study are asked to complete walk tests at home and to fill out weekly quality of life questionnaires using a mobile app, to enable the team to monitor how they are responding to the drugs.

They are monitored weekly during the trial for six months, but only four hospital visits - for MRI scans - are required throughout this time.

No study has ever put riociguat and selexipag ‘head-to-head’ in the UK in this way before, and Dr Varian believes the trial outcomes could have an impact of PAH care worldwide.

She said: “We really don’t know which drug is best. We feel like they might be equal in terms of certain parts of the way they improve PAH, but we may find for certain individuals one drug that suits them better than the other.

We hope the results will really help inform all care in the future when we think about prescribing these two therapies.

“There’s lots involved in the trial but at the end, each patient who takes part gets a personalised therapy. We don’t have to wait for the results of the full study, as we can make a decision based on their responses to the drugs, and any side effects.

It’s a really unique trial in that way because normally when you go into a trial, you don’t find out the results until years later. But because all the drugs in this trial are already approved, it’s all about how we can ensure the best therapy for the patient.”

The trial is currently running at the Sheffield Pulmonary Vascular Disease Unit and at Hammersmith Hospital in London, with plans to open it up around other UK specialist centres over the next two years.

The PHoenix study is focused on the treatment of PAH at this point, but there is significant potential for the findings to be used for patients with other forms of PH – and to influence the remote monitoring of patients in general.

“We’re in a fantastic era of change in terms of healthcare and there are so many new technologies that are coming through”, added Dr Varian.

“I think trials like this do offer a lot of hope and hopefully a better connection between the clinician and patient because of remote technology. I think all of these things are really exciting.”

• For more details about the PHoenix trial, please contact either the clinical trial manager Jenn Dick via email at jennifer.dick@sheffield.ac.uk or Dr Varian via email at f.varian@sheffield.ac.uk.

PHA UK members played a key role in shaping the design of the PHoenix trial by completing an important questionnaire in 2021. The survey, conducted by the PHA UK in collaboration with Dr Varian and her colleagues, asked questions around the perceived importance of the study, and its aims and objectives.

The responses helped secure funding for the trial, as well as shaping its design – a powerful example of the very real difference it makes when patients share their thoughts in this way.  Members of PHA UK are also present on the PHoenix steering committee.

Delivering a presentation at our Together Sheffield event in October (pictured below), Dr Varian spoke to the gathered patients, and their family and friends, about the importance of delivering patient-oriented research in this way.

 “The presentation was really a focus on how we can best design our research and clinical trials with the patient at the centre”, she said. “That means using all of the evidence that we have that’s been delivered through the questionnaires patients have completed in the past, interviewing them before the trial, and working with them throughout the study.

“We want to really have a partnership with them, and I have found that with PHoenix, patients have helped every step of the way with making sure we’re asking about side effects, medication burden, and quality of life regularly.

“Without having that dialogue with them, I don’t think the trial would be as well designed as it is now. It has really enhanced our study and the PHA UK is such a brilliant network to be able to engage with patients who are interested in being involved.”

Dr Varian also delivered a presentation about patient-oriented research at the National PH Research Forum, a gathering of PH researchers and clinicians organised by the PHA UK and held at the end of last year.  Aiming to inspire other researchers to keep patients at the centre of all studies, her presentation was voted the best of the conference, securing a £1,000 grant to enable her to further her work in this area.