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Gene therapy for haemophilia

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Abstract

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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.

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: 06 November 2014.

Selection criteria

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.

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 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.

PICOs

Population
Intervention
Comparison
Outcome

The PICO model is widely used and taught in evidence-based health care as a strategy for formulating questions and search strategies and for characterizing clinical studies or meta-analyses. PICO stands for four different potential components of a clinical question: Patient, Population or Problem; Intervention; Comparison; Outcome.

See more on using PICO in the Cochrane Handbook.

Plain language summary

Gene therapy for haemophilia

Haemophilia is a bleeding disorder caused by defective genes that produce abnormal blood clotting proteins. Gene therapy replaces these defective genes with normal ones. We looked for trials that used this approach of replacing defective genes for producing normal blood clotting proteins the treatment of haemophilia. As of 06 November 2014, 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.