Gene therapy for haemophilia

St Jude Children's Research Hospital, 262 Danny Thomas Place, MS 260, Memphis, Tennessee, USA, 38105. South Asian Cochrane Network & Center, Prof. BV Moses Center for Evidence-Informed Health Care and Health Policy, Christian Medical College, Carman Block II Floor, CMC Campus, Bagayam, Vellore, Tamil Nadu, India, 632002. QMed Knowledge Foundation, A-3, Shubham Centre, Cardinal Gracious Road,, Chakala, Andheri East, Mumbai, Maharashtra, India, 400099. Hematology Department, St Jude Children's Research Hospital, Hematology MS 800, Room R5006, 262 Danny Thomas Place, Memphis, Tennessee, USA, TN 38105-3678.

The Cochrane Database of Systematic Reviews. 2016;((12)):CD010822.
BACKGROUND Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy has recently been prompted as a curative treatment modality. This is an update of a published Cochrane Review. OBJECTIVES To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B. SEARCH METHODS We searched the Cochrane Cystic Fibrosis & Genetic Disorders Group's Coagulopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles and reviews.Date of last search: 18 August 2016. SELECTION CRITERIA Eligible trials include randomised or quasi-randomised clinical trials, including controlled clinical trials comparing gene therapy (with or without standard treatment) with standard treatment (factor replacement) or other 'curative' treatment such as stem cell transplantation for individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX. DATA COLLECTION AND ANALYSIS No trials of gene therapy for haemophilia were found. MAIN RESULTS No trials of gene therapy for haemophilia were identified. AUTHORS' CONCLUSIONS No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the safety and efficacy of gene therapy for haemophilia. Gene therapy for haemophilia is still in its nascent stages and there is a need for well-designed clinical trials to assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.
Study details
Study Design : Systematic Review
Language : English
Additional Material : Update in: ‘The Cochrane Database of Systematic Reviews’, (2020), PMID: 32342499, DOI: <a href=""></a>
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine