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Motor neuron disease (MND) is a devastating and debilitating disease with poor prognosis; most patients die from progressive respiratory failure within three years of onset. A randomised controlled trial conducted in Newcastle provided robust evidence that non-invasive ventilation for patients with MND can significantly improve quality of life and increase survival (216 days with non-invasive ventilation compared to 11 days without). Findings from this trial underpinned recommendations concerning the use of non-invasive ventilation in MND in clinical guidelines internationally, and use in clinical practice has increased in the UK, across Europe, and in the US and Australasia. In the UK, the number of MND patients successfully established on non-invasive ventilation in 2009 had increased 3.4-fold since 2000 and since 2009 has further increased almost two-fold.
Very considerable changes worldwide, in the management of patients with chronic obstructive pulmonary disease (COPD) admitted with acute ventilatory failure, have been a major impact of the research of the King's College London (KCL) Clinical Respiratory Physiology Group. Previously, invasive ventilation to treat such COPD patients was associated with complications which could be fatal. The KCL group conducted the first randomised controlled trial in the world of treatment by non-invasive ventilation (NIV). The trial demonstrated that NIV reduces complications and death compared to invasive ventilation. The study dramatically changed the treatment of hypercapnic flare-ups (where there is too much carbon dioxide in the blood) of acute COPD worldwide. NIV has become best and routine practice, and is advocated in national and international guidelines, benefiting many thousands of COPD patients.
In the 1960s boys with Duchenne muscular dystrophy would die at around the age of 14 to 15 years; by the 1990s survival had risen to around 19 years. Young men with this condition can now live to around 30 years of age. This significant improvement is possible where patient management involving coordinated multidisciplinary care is implemented. Such an approach was developed as a result of research and clinical practice pioneered by the Newcastle Muscle Group. Guidelines for the care of patients with Duchenne muscular dystrophy, published in 2010, were developed by an international working group led by Professor Kate Bushby of Newcastle University. These guidelines achieved NICE process accreditation in the UK and have been adopted globally as the definition of best practice.
Research at the University of Nottingham has defined the clinical phenotype and management of lymphangioleiomyomatosis, a rare and often fatal multisystem disease affecting 1 in 200,000 women worldwide. The group has led the development and evaluation of new therapies and diagnostic strategies which are now part of routine clinical care. The research has underpinned the transformation of this previously under recognised and untreatable disease into a condition recognised by respiratory physicians, with international clinical guidelines, patient registries, clinical trials, specific treatments and a UK specialist clinical service.
Neonatal extracorporeal membrane oxygenation (ECMO) is a complex procedure of life support used in severe but potentially reversible respiratory failure in newborn infants. In 1993 researchers in Leicester carried out the first and, to date, only large-scale randomised trial comparing the value of ECMO with other means of life support. The trial, with follow-up research at 4 and 7-year intervals, has shown ECMO to be a life-saving and cost-effective treatment, and has led to the establishment of a centrally funded neonatal programme that is estimated to have saved around 340 lives in the UK alone. In 2013 the University remains internationally renowned in the field of ECMO research, and since 2009 Glenfield Hospital has been home to the world's largest ECMO centre for the treatment of newborns, older babies and adults. The trial is still held up by advocates of fair clinical trials as an example of how evidence should translate into practice and policy.
Dialysis has revolutionised the management of End Stage Kidney Disease (ESKD), but the benefits of this invasive, demanding treatment may not be clear-cut for elderly, frail patients with other serious comorbidities. University of Hertfordshire and East and North Hertfordshire NHS Trust researchers have led the development of Conservative Management, an alternative to dialysis for some patients, providing multidisciplinary support and careful symptomatic management until death. The research shows that quality of life is maintained, survival may not be significantly compromised, and preferred place of death is more often achieved than for counterparts on dialysis. Conservative Management programmes have been adopted across the UK and elsewhere, influencing the care of many patients.
Three national, multicentre randomised controlled trials and associated studies during a 20-year research programme on abdominal aortic aneurysm (AAA) led by Imperial College researchers have altered international practice. The United Kingdom Small Aneurysm Trial (UKSAT) set the threshold for intervention at 5.5cm to repair AAAs. Population screening programmes and guidelines in Europe (UK, Sweden and Europe as a whole), Australia and the United States are based on these data. The results from EndoVascular abdominal Aortic Repair (EVAR) trials have informed international audits, guidelines (including NICE) and task forces in the same countries.
Professor Dickson's research group at Royal Holloway has pioneered the enabling technologies for the development of genetic therapies for the incurable disease Duchenne Muscular Dystrophy (DMD). Dickson's group has, (i) cloned replacement copies of the normal DMD gene, (ii) identified a natural substitute for the defective gene, and (iii) demonstrated that synthetic DNA can be used to correct the defective gene. The work has created impact on health and welfare through the development and clinical trials of a series of investigational medicinal products for this hitherto incurable disease, several clinical trials, and impact on commerce through industrial investment and licensed patents.
Research at Hull into hypersensitivity of the airways has provided novel insights into the epidemiology and causes of cough, and its burden on patients. This was achieved by the development of novel methodologies that allow the rigorous and objective testing of new and existing drugs. Patients benefit through the online provision of a diagnostic tool, and Proctor & Gamble have successfully exploited the cloned cell receptors in their drug development programme resulting in a new range of pharmaceuticals for cough. The work has underpinned the standardisation of cough challenge methodology through incorporation in national and international healthcare guidelines leading to a widespread improvement in patient treatment.
King's College London (KCL) research has had substantial impact, through making strong contributions to international and national guidelines which recommend that pulmonary rehabilitation should be made available to all appropriate people with COPD, including those who have had a recent hospitalisation for an acute flare-up.
Recommendations citing our research include those published internationally by a Cochrane Review and the Global Initiative for Chronic Obstructive Lung Disease, and nationally in the UK by NICE and IMPRESS — which provides clinical leadership to the NHS. These recommendations have been taken up by the NHS and are also included in NHS Best Practice Guidance (2012). The Department of Health (2010) estimated "widespread use of pulmonary rehabilitation would bring substantial annual savings".