Gene therapy for haemophilia.
BACKGROUND
Haemophilia is a genetic disorder which is 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.


OBJECTIVE
To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B.


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: 06 November 2014.


METHODS
Eligible trials included 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 individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX.


METHODS
No trials of gene therapy for haemophilia were found.


RESULTS
No trials of gene therapy for haemophilia were identified.


CONCLUSIONS
No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the effects 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.
Department of Pediatrics, University of Kentucky, 800 Rose Street, Room MN118, Lexington, Kentucky, USA, 40536.
Akshay
A
Sharma
Manu
M
Easow Mathew
Vasumathi
V
Sriganesh
Jessica A
JA
Neely
Sasank
S
Kalipatnapu
D015316:Genetic Therapy; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans