From a patient perspective, the concept of being involved in clinical research may be daunting, disconcerting, and unsettling but, equally, it can be rewarding to have the opportunity to increase the knowledge of how best to manage their condition. Congenital cardiology patients (those born with a heart condition) are often excluded from clinical trials and this means that whilst there is a vast experience-based knowledge of how to manage conditions and the complications related to them, there is not much ‘evidence-based medicine’ in clinical congenital cardiology. Much of how we manage non-congenital heart patients in later life is based around the results from large clinical trials where an active drug has been compared to a dummy drug, to demonstrate the effectiveness or not of the therapy. Over the last 30 years this has led to great advances in the management of heart failure and heart attacks, so that the treatment options for patients with these conditions are now established and highly effective. As a clinical community, it would be valuable for us to establish such evidence for treatments in the congenital cardiac patient.
The challenges to clinical research are numerous; it is a long and arduous process from the idea of a clinical trial through to the trial being approved, to patients being recruited and ultimately results being known that can guide clinical practice. Clinical care of patients should and does take priority over clinical research in all our institutions but increasingly there is a desire to take clinical research congenital cardiology research forward in the UK. The British Cardiovascular Society (BCS) and the National Institute for Health Research (NIHR) are keen to promote and support research in congenital cardiology. There is therefore an opportunity to establish the clinical research priorities in congenital cardiology, and the clinicians, nursing staff, patients and their families all have a role in developing patient research.
In the adult congenital heart disease (ACHD) service at Queen Elizabeth Hospital Birmingham (QEHB), we see large numbers of patients with highly complex congenital heart conditions, such as those with univentricular hearts who have undergone the Fontan operation in childhood, including an increasing number of patients with Hypoplastic Left Heart Syndrome, a very complex form of a univentricular circulation. Much of our clinical research programme is planned around the evaluation and long term management strategies for people with univentricular hearts.
We have recently evaluated the incidence of atrial arrhythmias in our population, demonstrating the high prevalence of rhythm disturbances in patients with the Fontan circulation. This information has led us to plan and perform some innovative electrophysiology techniques in these patients and to think how we can improve on our management strategy.
The evaluation of the functional capacity of univentricular heart patients is of great interest to our unit. We have used data from cardiopulmonary exercise testing to identify Fontan patients at risk of deterioration and are evaluating the use of 3D echocardiography techniques in assessing these patients. The aim of this is to lead us to a comprehensive non-invasive evaluation of patients to reduce the need for cardiac catheterisation and cardiac MRI and to identify patients who may require advanced management strategies.
Many of our patients would like to start a family and we are developing long term studies into the safety and outcomes of pregnancy in patients with congenital heart defects.
At QEHB, we will shortly open an Institute for Translational Medicine in which will be housed a Rare Diseases Unit. The unit is specifically set up to help clinical research in patient groups where the disease process is rare and the knowledge of how best to care for the patients is not fully developed. Virtually all of congenital heart disease falls under the ‘rare’ umbrella and we hope to start seeing all our transitional care patients within the unit, in order to fully evaluate their health status and general wellbeing, so that we can assess the long term impact of their heart condition on their life. Such studies are known as longitudinal studies, and do not necessarily require a drug intervention but do provide much needed information about the effects of childhood procedures, operations and interventions and can help in determining what are the best ways of treating children born with heart defects today.
So how can you become involved in clinical research? The most obvious answer is by participating in a clinical trial. Your hospital may be running a clinical trial looking at the management of your condition, and you may be asked to be involved. Typically, you will be given or sent a letter with an information sheet, detailing what the study involves; this should include the benefits of involvement and any risks that may be present. You will be asked to consent in writing to becoming a subject in the trial and it is worth knowing that even after you give consent, it is absolutely your choice should you wish to not be part of the trail later. Being in a trial will usually involve more clinical interaction, which may be for study investigations, blood tests, follow up appointments or in some cases in patient stays. You cannot be paid for being a participant, but many studies will reimburse any expenses incurred.
Another way of being involved is to review research proposals which a clinician may have developed. Increasingly, the bodies that pay for clinical research such NIHR require a panel of patients to review the research proposal to see that it makes sense to them and that the research could be of value to patients. This is important as it is all of us who pay for research through our taxation and charitable contributions and the funders need to know that their money is spent wisely.
Finally, it is recognised that many of the best ideas for research come from patients themselves, and the BCS are hoping to survey patient groups and clinicians in the near future to hear what they think the priorities for clinical research should be. Hopefully this will pull together research themes which again will help the funders in determining what research is most likely to benefit patients.
If you have any good ideas for clinical research, please do feed them back to The Somerville Foundation and they will be taken seriously. If in the future you are sent a survey to complete then we really do want to know your views on clinical research and also to hear about your experiences, both good and bad, of clinical research.
All of us involved in clinical research are immensely grateful to patients and their families for the time and commitment that they give to clinical studies. Without you, clinical research is simply not possible, but with you there is a great potential to develop new and effective management strategies for patients with congenital heart defects, and we thank you for your continued support.
By Paul Clift
Consultant Cardiologist QEHB, Clinical Lead for NIHR, Chair of BCS Clinical Study Group for Congenital Research