6 November - A new drug for patients with a severe and rare form of anaemia associated with chronic kidney disease may be more effective than time-consuming and expensive blood transfusions, research carried out at King’s College Hospital has shown.
The results of the clinical drug-trial at King’s – which are reported in this week’s edition of the New England Journal of Medicine (NEJM) – support the use of a novel and highly effective treatment for pure-red cell aplasia (PRCA), a form of anaemia in patients with chronic kidney disease which causes a sudden and severe decrease in the number of red blood cells produced by the bone marrow.
At present, the only recognised treatment for patients with the condition is regular blood transfusions, or alternatively powerful immunosuppressive drugs which can expose the patient to an increased risk of infections. The effectiveness of blood transfusions is limited, and they also carry a high risk of infection. Blood transfusions can also cause the body to develop antibodies which may complicate a later kidney transplant.
The trial at King’s involved giving monthly injections of Hematide (an experimental erythropoiesis-stimulating agent) to 14 patients with PRCA over a mean period of two years. All but one of the patients experienced an increase in red blood cell production, meaning blood transfusions were not needed.
Many patients with chronic kidney disease develop anaemia. This is because the failing kidneys struggle to produce enough erythropoietin, a hormone crucial to the process of red blood cell production. Many patients thus become anaemic, which can cause shortness of breath, loss of energy, and exacerbate problems associated within chronic kidney failure. These patients, for the last 20 years, have received erythropoietin replacement therapy for their anaemia.
Pure-red cell aplasia is a particularly severe form of the condition, which develops in 1 in 10,000 patients receiving erythropoietin therapy, in which the patients produce antibodies against their treatment. These antibodies not only abolish the effect of their erythropoietin therapy, but also completely shut down red cell production by the bone marrow, resulting in many patients feeling chronically exhausted and requiring blood transfusions every two weeks long-term.
Dr Iain Macdougall, consultant nephrologist at King’s and the lead investigator for the study, said:
“For the first time, we have a treatment that (once licensed) can rescue these severely debilitated patients from a lifetime of misery and dependence on blood transfusions. Prior to this, doctors were desperately using heavy immunosuppressive drugs to get rid of these antibodies, and exposing the patient to an increased risk of life-threatening infections. Many patients with this condition previously died”.
This trial is an example of how King’s, as well as King’s College London, and Guy’s and St Thomas’ and South London and Maudsley NHS Foundation Trusts (all members of King’s Health Partners Academic Health Sciences Centre), are leading cutting-edge research to improve patient care. King’s Health Partners is one of only five accredited AHSCs in the UK and is a collaboration designed to reduce the time it takes for research discoveries and medical breakthroughs to become routine clinical practice.
The research paper, A Peptide-Based Erythropoietin-Receptor
Agonist for Pure Red-Cell Aplasia, was published in this week’s issue of the New England Journal of Medicine (NEJM).
Notes to editors
Patient case study available on request.
1. For media enquiries only contact:
Chris Rolfe, Corporate Communications,
King’s College Hospital NHS Foundation Trust.
Tel: 020 3299 3006, Fax: 020 3299 3207
e-mail: chris.rolfe@kch.nhs.uk
www.kch.nhs.uk
2. King’s College Hospital NHS Foundation Trust is one of the UK’s largest and busiest teaching hospitals, with over 6,600 staff providing around 800,000 patient contacts a year. King’s has a unique profile, with a full range of local hospital services for people in the London boroughs of Lambeth and Southwark as well as specialist services to patients from further afield. The Trust is recognized internationally for its work in liver disease and transplantation, neurosciences, cardiac and haemato-oncology. King’s also plays a key role in the training and education of medical, nursing and dental students with its academic partner, King’s College London. For more information, visit www.kch.nhs.uk
3. King’s College Hospital NHS Foundation Trust is part of King’s Health Partners Academic Health Sciences Centre (AHSC), a pioneering collaboration between King’s College London, and Guy’s and St Thomas’, King’s College Hospital and South London and Maudsley NHS Foundation Trusts.
King’s Health Partners is one of only five AHSCs in the UK and brings together an unrivalled range and depth of clinical and research expertise, spanning both physical and mental health. Our combined strengths will drive improvements in care for patients, allowing them to benefit from breakthroughs in medical science and receive leading edge treatment at the earliest possible opportunity.
For more information, visit www.kingshealthpartners.org
