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We reviewed the evidence about the safety and effectiveness of gene therapy for treating people with haemophilia A or haemophilia B.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2017

In haemophilia and other congenital bleeding disorders blood does not clot properly, which can cause excessive bleeding. This is particularly relevant during surgery, when the risk of bleeding depends on the type and severity of the clotting disorder and on the type of surgery. Therefore, during and after surgery, these individuals should receive treatment to improve the ability of their blood to clot and so prevent bleeding. Clotting factor concentrates (when available and appropriate in those individuals missing specific clotting proteins) or other non‐specific drugs for clotting, or a combination of both, are administered. It is not known what is the optimal dose or duration or method of administration of these treatments in these circumstances.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

We reviewed evidence about the effect of immune tolerance induction to remove inhibitors in people with haemophilia A and B.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

Hemophilia A and B are X‐linked inherited bleeding disorders, in which the major clinical problem is repeated bleeding into joints. As this disorder progresses, joints become deformed and movement limited. Current therapy for treating and preventing bleeding includes plasma‐derived or recombinant clotting factor concentrates. This review includes six randomised controlled trials. Two compare the regular use of clotting factor concentrates to prevent joint bleeds with their use 'on demand'. Four compare different regimens of regular use in children and adults with hemophilia. It was clearly evident that preventative therapy, as intravenous infusion of factor concentrate repeated more times a week and started early in childhood was able to reduce joint deterioration as compared to treatment administered after bleeding occurred. This favourable effect is due to a consistent reduction in total bleeds and hemarthrosis (bleeding into joints) and leads to a significant improvement in quality of life. Preventative therapy is linked to an increased factor usage and cost of treatment. We found weaker evidence (due to lack of data) to show preventative therapy reduced joint deterioration when treatment is started after joint damage has been established. Further studies are needed to establish the best preventative regimen, i.e. for example starting time, dosage frequency, minimally effective dose.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2011

We reviewed the evidence about the safety and usefulness of exercise for men with haemophilia.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2017

We wanted to find evidence on the effectiveness of recombinant factor VIIa (containing no human proteins) as compared to concentrates derived from plasma for treating acute bleeding episodes in people with haemophilia with inhibitors.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

We reviewed the evidence about whether antifibrinolytic medicine (drugs that promote blood clotting) such as tranexamic acid or epsilon aminocaproic acid, can prevent oral bleeding in people with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2016

People who have severe hemophilia A may benefit from regularly taking clotting factor medication as preventive treatment. This may lower the risk of severe bleeding.

Informed Health Online [Internet] - Institute for Quality and Efficiency in Health Care (IQWiG).

Version: March 9, 2016

In the last three decades there have been dramatic improvements in the availability and quality of treatment for people with inherited coagulation disorders. Indeed, the improvement of methods of purification and viral inactivation for plasma-derived coagulation factor concentrates first and then the development of products utilizing recombinant DNA technology have greatly improved the life expectancy of hemophiliacs, which has progressively become similar to that of males in the general population. Nowadays, the most frequent complication of factor replacement therapy for hemophilia is the development of inhibitors. However, no studies so far have systematically analysed the type and incidence of other adverse reactions following the administration of coagulation factor concentrates. The aim of this systematic review was to screen the published literature data to evaluate the types and frequencies of non-thrombotic-, non-inhibitor-associated adverse reactions to coagulation factor concentrates in patients with hemophilia A, hemophilia B and von Willebrand's disease. On behalf the European Haemophilia Safety Surveillance System (EUHASS), a systematic review of the prospective studies published in the last 20 years was performed using electronic databases and article references. Both severe and mild adverse events following infusion of coagulation factor concentrates are relatively rare in patients with inherited coagulation disorders; the most common events are of an allergic type. There are no differences in the rate of adverse events caused by plasma-derived or recombinant products. On the whole, these data confirm the high degree of safety of the products currently used for replacement therapy.

Database of Abstracts of Reviews of Effects (DARE): Quality-assessed Reviews [Internet] - Centre for Reviews and Dissemination (UK).

Version: 2012

Thrombotic adverse events (AEs) after clotting factor concentrate administration are rare but the actual rate is unknown. A systematic review of prospective studies (1990-2011) reporting safety data of factor concentrates in patients with haemophilia A (HA), haemophilia B (HB) and von Willebrand disease (VWD) was conducted to identify the incidence and type of thrombotic AEs. In 71 studies (45 in HA, 15 HB, 11 VWD) enrolling 5528 patients treated with 27 different concentrates (20 plasma-derived, 7 recombinant), 20 thrombotic AEs (2 HA, 11 HB, 7 VWD) were reported, including two major venous thromboembolic episodes (both in VWD patients on prolonged replacement for surgery). The remaining thrombotic AEs were superficial thrombophlebitis, mostly occurring at infusion sites in surgical patients and/or during concentrate continuous infusion. The overall prevalence was 3.6 per 10(3) patients (3.6 per 10(4) for severe AEs) and 1.13 per 10(5) infusions, with higher figures in VWD than in haemophilia. Thrombotic AEs accounted for 1.9% of non-inhibitor-related AEs. Thrombosis-related complications occurred in 10.8% of patients with central venous access devices (CVADs) reported in six studies, the risk increasing with time of CVAD use. Data from prospective studies over the last 20 years suggest that the risk of thrombotic AEs from factor concentrate administration is small and mainly represented by superficial thrombophlebitis. These findings support the high degree of safety of products currently used for replacement treatment.

Database of Abstracts of Reviews of Effects (DARE): Quality-assessed Reviews [Internet] - Centre for Reviews and Dissemination (UK).

Version: 2012

Hemophilia is a disease that causes problems with blood clotting. It makes people's blood clot (coagulate) much more slowly than usual. The more poorly the blood clots, the worse the symptoms are. Read about the degrees of severity, the causes and effects of hemophilia, and the treatment options.

Informed Health Online [Internet] - Institute for Quality and Efficiency in Health Care (IQWiG).

Version: March 10, 2016

This study reviewed evidence on group clinics (shared consultations for people with chronic disease). Most studies focused on diabetes and found that group clinics were better than individual appointments for some clinical markers, but not across all outcomes. Most studies took place in the USA and may not be applicable to a UK context, so more research is needed to find out if group clinics would be acceptable and value for money in the UK NHS.

Health Services and Delivery Research - NIHR Journals Library.

Version: December 2015

We reviewed the evidence available to see if rituximab is effective and safe when treating clotting factor inhibitors in people with severe hemophilia. This is an update of a previously published Cochrane Review.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2017

This report evaluates the level of evidence currently available to support the effectiveness and safety of using recombinant activated coagulation factor VII (rFVIIa) for clinical indications not approved by the U. S. Food and Drug Administration (FDA). rFVIIa is approved for a variety of uses in hemophilia patients who have developed antibody inhibitors that compromise the use of standard factor replacement. Use of this costly biologic product has expanded beyond these hemophilia-related indications to encompass a range of off-label uses, most of which are in-hospital uses. These uses differ substantially from the drug’s FDA approved label. The purpose of this report is two-fold: (1) To document the full range of clinical indications for which rFVIIa is being used and the types of studies available to evaluate these uses and (2) To provide a comparative effectiveness review of rFVIIa vs. usual care for several in-hospital clinical indications: intracranial hemorrhage, massive bleeding secondary to trauma, and the selected surgical procedures of cardiac surgery, liver transplantation, and prostatectomy.

Comparative Effectiveness Reviews - Agency for Healthcare Research and Quality (US).

Version: May 2010

Venous thromboembolism (VTE) is a condition in which a blood clot (a thrombus) forms in a vein and then dislodges to travel in the blood (an embolus). A venous thrombus most commonly occurs in the deep veins of the legs or pelvis; this is then called a deep vein thrombosis (DVT). Blood flow through the affected vein can be limited by the clot, and it can cause swelling and pain in the leg. If it dislodges and travels to the lungs, to the pulmonary arteries, it is called a pulmonary embolism (PE), which in some cases may be fatal. VTE as a term includes both DVT and PE. Major risk factors for VTE include a prior history of DVT, age over 60 years, surgery, obesity, prolonged travel, acute medical illness, cancer, immobility, thrombophilia (an abnormal tendency for the blood to clot) and pregnancy.

NICE Clinical Guidelines - National Clinical Guideline Centre (UK).

Version: June 2012

Infusion of bypassing agents, such as recombinant activated FVII (rFVIIa) and plasma‐derived activated prothrombin complex concentrate (APCC), are suggested as alternative therapies to factor VIII (haemophilia A) or IX (haemophilia B) for individuals who no longer respond to these treatments because they develop inhibitory antibodies. The ultimate goal of treatment is to preserve the individual's joints, otherwise destroyed by recurrent bleeds. We therefore evaluated the effectiveness and safety of bypassing agents when used to prevent, as compared to treat, bleeds. We also compared different doses of bypassing agents in men with hemophilia A or B with inhibitors as a preventative (prophylactic) therapy.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2017

Study found that ivacaftor is a clinically effective treatment for patients with cystic fibrosis and the G551D mutation; however, the high cost of ivacaftor may be a barrier to its uptake. Ivacaftor resulted in significant improvements compared with placebo for all but one of the subgroups investigated; further research should prioritise investigating its long-term effectiveness.

Health Technology Assessment - NIHR Journals Library.

Version: March 2014

This review evaluated the effectiveness of treatments for acute bleeding in patients with haemophilia A with inhibitors. The authors concluded that bleeding control varied from 60 to 100%. The authors undertook a systematic search but the lack of detail on review methodology and the poor quality of many included studies make the reliability of the conclusions uncertain.

Database of Abstracts of Reviews of Effects (DARE): Quality-assessed Reviews [Internet] - Centre for Reviews and Dissemination (UK).

Version: 2003

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