Authors' objectives: Beta-thalassaemia major is an inherited blood disorder that affects red blood cells. Red blood cells contain haemoglobin, a substance that carries oxygen from the lungs to the rest of the body. People with beta-thalassaemia major have a fault (or mutation) on a gene (the beta-globin gene) and so make abnormal haemoglobin that does not function normally. This means that people with beta-thalassaemia major have ineffective red blood cells, and they need to have blood transfusions throughout their life. The frequency of blood transfusions can vary among patients but most people typically have blood transfusions every 3-4 weeks. Treatment of beta-thalassaemia major with transfusions can cause too much iron to build up in the body and this can cause many complications. Drugs called iron-chelators are used to remove excess iron from the body, but these drugs can be difficult to take and may require patients to carry a pump for many hours at a time to deliver drugs directly under the skin. Some patients can be cured if they receive a stem cell (bone marrow) transplant, but this is only possible in a small number of patients who have a matching donor. LentiGlobin BB305 is a new gene therapy for the treatment of beta-thalassaemia major. It is delivered as a single dose straight into the blood following a course of chemotherapy. If LentiGlobin BB305 is licenced for use in the UK, it could be the first treatment option for beta-thalassaemia major that has the potential to add a working copy of the beta-globin gene to patients' stem cells. It may improve survival and quality of life by reducing or eliminating the need for blood transfusions and iron-chelation therapy.

Project Status: Completed

Year Published: 2016

URL for published report: http://www.hsric.nihr.ac.uk/topics/lentiglobin-bb305-lentiglobin-for-beta-thalassaemia-major-first-line/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)