Similar case studies

REF impact found 144 Case Studies

Currently displayed text from case study:

Use of non-invasive ventilation to improve survival and quality of life in patients with motor neuron disease

Summary of the impact

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.

Submitting Institution

Newcastle University

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Cardiorespiratory Medicine and Haematology, Clinical Sciences, Neurosciences

Diagnosis and treatment of congenital myasthenic syndrome in patients with Dok-7 mutations

Summary of the impact

Congenital myasthenic syndromes (CMS) are inherited neuromuscular disorders caused by defects at neuromuscular junctions, which are often a result of acetylcholine receptor gene mutations. A subset of CMS patients (around 14% in the US and Europe) have limb-girdle myasthenia (LGM). This disease can be highly disabling with symptoms including increasing weakness of skeletal muscles. As a result of collaborative work between Newcastle and Oxford, it was determined that many LGM patients have a mutation of the Dok-7 gene (unrelated to the acetylholine receptor), and do not, therefore, respond to standard CMS treatments. Since then, a number of additional mutations have been discovered, and genetic testing is now available for the majority of known LGM-causative genes. Crucially, Dok-7 patients, and those with other non-receptor related mutations, can now be diagnosed accurately and treated effectively, with ephedrine and salbutamol (in the US, albuterol). This significantly improves these patients' quality of life by enabling them to walk and breathe unassisted.

Submitting Institution

Newcastle University

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Health

Research Subject Area(s)

Biological Sciences: Genetics
Medical and Health Sciences: Neurosciences

The development of psychological treatment pathways and better identification of Medically Unexplained Symptoms (MUS) in primary care

Summary of the impact

Work on better management and identification of Medically Unexplained Symptoms (MUS) has led to the introduction of new treatments in primary care in England through the Improving Access to Psychological Treatment (IAPT) programme as well as having an impact on service planning and commissioning. These approaches have also been implemented into the routine training and practice of General Practitioners (GPs) in parts of Europe. A clinical and economic evaluation of a psychosocial approach to chronic fatigue syndrome using general nurses and development of a cognitive behaviour therapy approach has changed general practice and enhanced the patient experience for those with MUS.

Submitting Institution

University of Nottingham

Unit of Assessment

Psychology, Psychiatry and Neuroscience

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Public Health and Health Services

Kinetic modelling to optimise dialysis and improve kidney function in patients with multiple myeloma

Summary of the impact

Through the mathematical modelling of biomedical processes, outcomes for patients with multiple myeloma (a form of bone cancer that can lead to kidney failure, a major cause of morbidity) have been vastly improved, with the result that dialysis treatment may no longer be necessary alongside their myeloma treatment. Regaining kidney function for these patients improves their quality of life and results in significant cost savings for the NHS. Research into the modelling of the kinetics of whole antibodies produced by multiple myeloma patients (and others) has also had an impact on patient outcomes and clinical decisions. There have also been economic benefits for Gambro, a German company that sells products developed on the basis of the research results.

Submitting Institution

University of Warwick

Unit of Assessment

General Engineering

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences

Improving clinical care for lymphangioleiomyomatosis

Summary of the impact

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.

Submitting Institution

University of Nottingham

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Cardiorespiratory Medicine and Haematology, Clinical Sciences, Public Health and Health Services

Clinical development and manufacture of a new drug, ChronocortĀ®, for treatment of the rare orphan disease congenital adrenal hyperplasia

Summary of the impact

Research on Congenital Adrenal Hyperplasia (CAH) at the University of Sheffield has resulted in both health and commercial impacts. The research has led to a new drug treatment, Chronocort®, being developed for CAH. Chronocort® has been tested in CAH patients with the positive outcome of improved disease control.

Commercial impact arose from the creation of a spin-out company, Diurnal Ltd, in 2004 which has raised investment of £3.8M since 2008, including £0.4M from pharmaceutical industry sources, and (as an SME partner) a €5.6M Framework 7 grant to develop a paediatric treatment for CAH. Diurnal has created five new jobs and has contracts with six UK companies worth £2.7M.

Submitting Institution

University of Sheffield

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences, Paediatrics and Reproductive Medicine

A new gold standard treatment for the emergency correction of warfarin-induced coagulopathy

Summary of the impact

As a result of University of Sheffield research in 1995-2002, a new gold standard treatment for major bleeding on warfarin has been established, ensuring the more effective treatment of tens of thousands of patients requiring emergency anticoagulation reversal each year in the UK alone. The treatment, using prothrombin complex concentrate (PCC) was demonstrated to be superior to fresh frozen plasma (FFP), the standard alternative at the time, and two PCCs have now been licensed for this indication in the UK.

UK and international guidelines now recommend PCC over FFP.

Submitting Institution

University of Sheffield

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Cardiorespiratory Medicine and Haematology, Clinical Sciences

Uncovering the genetic basis of atypical haemolytic uraemic syndrome leads to improved treatment.

Summary of the impact

Research conducted by Professor Tim Goodship and co-workers at Newcastle has had a profound effect on the prognosis for patients with atypical haemolytic uraemic syndrome (aHUS). By engaging in research on the genetic factors underlying the disease they developed an understanding of the molecular mechanisms responsible. Identifying that the majority of patients with aHUS have either acquired or inherited abnormalities of the regulation of complement (part of the immune system) led to the establishment of a UK national service for genetic screening and treatment with the complement inhibitor eculizumab. As eculizumab is now available to patients in England, the progression to end-stage renal failure can be prevented and patients already on dialysis will soon be successfully transplanted.

Submitting Institution

Newcastle University

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Clinical Sciences

Influencing national policy to improve service delivery and patient care in gastroenterology

Summary of the impact

Between 1996 and 2013 researchers at Swansea University evaluated service initiatives and changing professional roles associated with the management of patients with debilitating gastrointestinal disorders. This work showed the clinical and cost effectiveness of two main innovations: open access to hospital services for patients with inflammatory bowel disease; and increased responsibility for nurses, particularly as endoscopists. Our evidence has had a broad, significant impact on: national policy through incorporation in NHS strategies, professional service standards and commissioning guides; service delivery through the provision of increasing numbers of nurse endoscopists and the wide introduction of nurse-led open access to follow-up; and patient care, as documented in sequential national audits in 2006, 2008 and 2010.

Submitting Institution

Swansea University

Unit of Assessment

Allied Health Professions, Dentistry, Nursing and Pharmacy

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Nursing, Public Health and Health Services

How better risk stratification for lung transplant has benefitted cystic fibrosis patients

Summary of the impact

Lung transplants represent the last hope for cystic fibrosis patients with end-stage lung disease. However, since the mid-1990s, other than in large research centres, some cystic fibrosis patients were not offered this treatment because of the variable and often poor outcome of surgery. This patient group carried a difficult to treat bacterial infection caused by the Burkholderia genus. In 2001 researchers in Newcastle published findings that demonstrated that one particular species, Burkholderia cenocepacia, was responsible for the poor outcomes and that other species of Burkholderia were not as dangerous. This finding was incorporated into international guidelines and since 2008 most transplant centres worldwide have adopted a risk stratification approach to listing patients for transplant. Consequently, more than 30 people per year worldwide now get transplants that would otherwise have been denied.

Submitting Institution

Newcastle University

Unit of Assessment

Clinical Medicine

Summary Impact Type

Health

Research Subject Area(s)

Medical and Health Sciences: Cardiorespiratory Medicine and Haematology, Paediatrics and Reproductive Medicine

Filter Impact Case Studies

Download Impact Case Studies