GSK2696273 for severe combined immunodeficiency due to adenosine deaminase deficiency
NIHR HSRIC
Record ID 32016000384
English
Authors' objectives:
GSK2696273 is a gene therapy intended for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) in patients for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
ADA-SCID accounts for about 10-15% of all cases of severe combined immunodeficiency. An estimated 14 children are born with the condition every year in Europe, and the annual incidence is estimated to be between 1 in 200,000 and 1 in 1,000,000 live births. In 2013-14, there were 190 admissions for adenosine deaminase deficiency in England, resulting in 531 bed days and 205 finished consultant episodes.
Patients with ADA-SCID are initially treated with antibiotics, antiviral and antifungal medicines, intravenous immunoglobulins and prophylaxis for Pneumocystis jiroveci, but most ultimately require a bone marrow transplant. The recommended treatment is based on allogeneic haematological stem cell transplantation (HSCT) from an HLA identical healthy sibling or close relative, which has a high success rate. However, most patients lack an identical donor. Other treatment options include HSCT from an HLA matched unrelated donor, an HLA haploidentical donor (usually a parent) or umbilical cord derived stem cells; enzyme replacement therapy with pegylated adenosine deaminase enzyme; or gene therapy (currently experimental).
Currently, GSK2696273 is in a single arm phase II/III clinical trial studying its effects on overall survival. This trial is expected to complete in June 2019.
Details
Project Status:
Completed
Year Published:
2015
URL for published report:
http://www.hsric.nihr.ac.uk/topics/gsk2696273-for-severe-combined-immunodeficiency-due-to-adenosine-deaminase-deficiency/
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
England, United Kingdom
MeSH Terms
- Infant, Newborn, Diseases
- Adenosine Deaminase
- Agammaglobulinemia
- Severe Combined Immunodeficiency
- Genetic Therapy
Contact
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)
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