Research highlights need to address hemophilia in developing world

November 16, 2009

When modern medicine finds a way to treat a medical condition, people often think that the problem is solved. But we also have to find ways to get that treatment into the hands of those who need it. For example, new research from North Carolina State University shows that much more needs to be done to help get existing treatment to hemophilia patients in the developing world, and that the current lack of treatment there is costing lives.

"This research illustrates international disparities in treatment for a disease that we know how to address," says Dr. Jeff Stonebraker, an assistant professor of business management at NC State and lead author of two new studies on hemophilia prevalence and treatment. "What we've found highlights the work that needs to be done by governments, health officials and pharmaceutical manufacturers to address the needs of those suffering with hemophilia in the developing world."

Type A hemophilia is a hereditary bleeding disorder that affects about 400,000 people - predominantly men - around the world. The disorder prevents blood from clotting normally, creating the risk of serious bleeding or internal bleeding. Ultimately, the disorder can result in severe pain, joint deformities and death in childhood or young adulthood. But the condition can be treated by replacing the missing clotting factor VIII, which enables those suffering from hemophilia A to live relatively normal lives. Unfortunately, the treatment is expensive and new research shows that access to that treatment is severely limited.

"This is the best data there is on a relatively small global population," says Mark Skinner, president of the World Federation of Hemophilia. "It will be incredibly useful to our members, to manufacturers of treatment products, ministries of health and researchers. These two very important papers help us see where progress is being made and how we can learn lessons that will help us continue to improve care and work toward our goal of treatment for all."

In the first study, the researchers found that prevalence of hemophilia A in high-income countries was approximately 12.8 per 100,000 males. The prevalence in lower-income countries was approximately 6.6 per 100,000 males. "The medical community tells us that the incidence of hemophilia A - or the number of people born with the condition - is the same around the world," Stonebraker explains, "so the difference in prevalence - or the number of people living with the condition at any given moment - appears to be due to much higher mortality in developing countries."

The study also showed that prevalence of hemophilia has increased over the past 30 years, as treatments for the disorder have improved. In other words, better treatment is helping those with hemophilia A live longer. For example, the United Kingdom had a prevalence of 9.3 per 100,000 in 1974, but it had risen to 21.6 per 100,000 by 2006.

In a second study, Stonebraker and his colleagues found that decreased mortality related to hemophilia A is tied to a willingness - by government health-care agencies or private insurers - to pay for treatment. Correspondingly, the consumption of factor VIII drugs has increased significantly in developed countries - and that trend appears poised to continue, with high-income countries expected to consume more and more factor VIII drugs in the future.

Stonebraker says the two studies should be incredibly helpful as budget-planning tools for insurance companies and those countries that provide national health care, as well as for pharmaceutical companies that will want to project the amount of factor VIII drugs they will need to make to meet market demand. But, Stonebraker says, "the studies also show how much more needs to be done to address hemophilia in the developing world."
-end-
The first study, "A study of variations in the reported hemophilia A prevalence around the world," was co-authored by Stonebraker, Paula H.B. Bolton-Maggs of the Manchester Royal Infirmary, J. Michael Soucie of the U.S. Centers for Disease Control and Prevention, Irwin Walker of McMaster University and Mark Brooker of the World Federation of Hemophilia. The second study, "A study of reported factor VIII use around the world," was co-authored by Stonebraker, Brooker, Robert E. Amand of the Biotherapeutic Modeling Group, Inc., Albert Farrugia of the Plasma Protein Therapeutics Association and Alok Srivastava of Christian Medical College. Both studies will be published in a forthcoming issue of Haemophilia.

North Carolina State University

Related Hemophilia Articles from Brightsurf:

A new cell & gene therapy approach to treat common bleeding disorder
WFIRM researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.

Early treatment linked to reduced joint damage in young adults with severe hemophilia A
Children with severe hemophilia A who receive early prophylaxis are less likely to have joint damage in young adulthood than those who begin treatment later, according to a new study in Blood Advances.

Hemophilia three times more prevalent than thought
For the World Federation of Hemophilia, Iorio assembled an international team of researchers from France, US and UK to perform a meta-analysis of the registry data in countries with the most comprehensive registries of hemophilia, which were Australia, Canada, France, Italy, New Zealand and the UK.

Gene-based factor VIIa prevents bleeding episodes in animals with hemophilia
Hematology researchers have further refined how a treatment currently used on an urgent basis to control bleeding in hemophilia patients holds promise as a preventive treatment as well.

Single injection treats hemophilia B for life, in proof-of-concept study
Salk researchers have demonstrated in mice that hemophilia B can be treated for life with one single injection containing disease-free liver cells that can produce their missing clotting factor.

Groundbreaking gene therapy trial set to cure hemophilia
A 'cure' for hemophilia is one step closer, following results of a groundbreaking gene therapy trial led by Queen Mary University of London and the NHS in London.

Inflammatory factors linked to inhibition of factor VIII gene therapy in hemophilia A
As a gene therapy cure for hemophilia A moves closer to reality, a new study sheds light on a challenging complication in which a host autoimmune response inhibits the production of normal clotting factor VIII from the transferred FVIII gene.

Spark Therapeutics and Pfizer announce publication in The New England Journal of Medicine of Interim Data from phase 1/2 clinical trial of investigational gene therapy for Hemophilia B
Spark Therapeutics (NASDAQ: ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer Inc.

Disruptive technology for the treatment of hemophilia
An international team of hematologists including Guy Young, MD, of Children's Hospital Los Angeles, has found that in patients with hemophilia A with inhibitors, a novel therapy called emicizumab, decreases incidence of bleeding episodes by 87 percent.

Researchers improve vbectors for delivering hFVIII gene therapy to treat Hemophilia A
A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward into development.

Read More: Hemophilia News and Hemophilia Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.