Review question
We reviewed the evidence about the safety and effectiveness of gene therapy for treating people with haemophilia A or haemophilia B.
Background
Haemophilia is a bleeding disorder caused by defective genes that produce abnormal blood clotting proteins. Gene therapy modifies or replaces these defective genes with normal ones. We looked for trials that used this approach of modifying or replacing defective genes for producing normal blood clotting proteins for the treatment of haemophilia. This is an update of a published Cochrane Review.
Search date
The evidence is current to: 17 April 2020.
Key results
We found no trials to provide reliable evidence about the risks or benefits of gene therapy for haemophilia. There is a need for trials that assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.
Read the full abstract
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 for haemophilia is a curative treatment modality currently under investigation. 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 strategy
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: 17 April 2020.
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 matching the inclusion criteria were identified.
Main results
No trials of gene therapy for haemophilia matching the inclusion criteria 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 clinical investigation 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.
