Hemophilia World Day: One Family's Story
April 17th marks World Hemophilia Day 2012, when the international bleeding disorders community will come together to help spread the message to "Close the Gap" and to improve accessibility and quality of care so that effective treatment will be available for all around the world.
About Hemophilia and Other Bleeding Disorders
Hemophilia, von Willebrand disease, and other inherited clotting factor deficiencies are lifelong bleeding disorders that prevent blood from clotting properly. People with bleeding disorders either do not have enough of a particular clotting factor, a protein in blood that controls bleeding, or their clotting factor does not work properly. Bleeding into joints and muscles causes severe pain, chronic joint disease and disability and bleeding into major organs, such as the brain, can cause death.
People with hemophilia use treatment products to improve blood clotting and stop or prevent a bleeding episode. People with hemophilia have a higher quality of life today than ever before, but complications can still occur. As many as 15-20% of people with hemophilia will develop an antibody—called an inhibitor—to the product used to treat or prevent bleeding episodes. The inhibitors stop the treatment product from working. This makes it more difficult to stop a bleeding episode. Developing an inhibitor is one of the most serious and costly complications of hemophilia.
One Family's Story
"Leland has hemophilia. At 15 months old, Leland developed a complication called an inhibitor. This means that his body, his immune system, fights off the medicine – the infused factor VIII. That, quite honestly, pulled the rug out from underneath us, when the preferred methods of treatment were no longer options for us. The alternative clotting factors that we use to control bleeding are much less effective. His treatment more closely resembles that which was done a generation ago; he doesn't benefit from a lot of the advances in treatment that help his peers who don't have an inhibitor.
The complications of long-term damage to his joints, the unrelenting pain, just general quality of life, that's been by far the biggest challenge. Another big challenge for us as a family and, for my husband in particular, is the high cost of hemophilia in general and treating an inhibitor in particular. My husband has had to make career choices based on maintaining good health insurance.
Having a son with hemophilia and then, the added challenge of an inhibitor, is not always easy. As a parent, I would love to fix things and make everything better. The inhibitor has taken away a lot of the control that parents have in effectively managing hemophilia. We don't really have a proactive management of the bleeding; we have to wait until bleeds happen, and then deal with them from there. I've watched him sit out and miss a lot of important life experiences, including most of high school. It's not what I envisioned when he was a baby and first diagnosed.
Fortunately, there have been many other positive life experiences that have come from having hemophilia. He's been in Washington advocating to his legislators, he's a regular guest speaker at Harvard Medical School, and he's traveled around the country for community events. How many teenagers get to do that? Hopefully these experiences help maintain some balance, for both Leland and our family as a whole."
Comprehensive Treatment Centers
CDC's National Center on Birth Defects and Developmental Disabilities, along with other federal agencies, supports a network of comprehensive treatment centers able to meet the unique challenges of people with hemophilia and other bleeding disorders, including von Willebrand Disease and other clotting factor deficiencies. Comprehensive treatment centers are specialized healthcare settings that bring together a team of doctors, nurses, and other health professionals experienced in treating people with rare or complex chronic medical conditions.
A CDC study of 3,000 people with hemophilia conducted in six U.S. states showed that those who received care in a hemophilia treatment center were 40% less likely to die of a hemophilia-related complication compared to those who did not receive care at a treatment center. Similarly, people who used a treatment center were 40% less likely to be hospitalized for bleeding complications.
Each hemophilia treatment center provides access to multidisciplinary health care professionals:
- Hematologists (doctors who specialize in blood)
- Orthopedists (doctors who specialize in bones, joints, and muscles)
- Physical therapists
- Social workers and other mental health professionals
Comprehensive hemophilia treatment centers emphasize prevention services to help reduce or eliminate complications. These services include using preventive medicine and connecting patients with community groups that provide education and support to families.
CDC also supports treatment and research center networks for other bleeding and clotting disorders. There are treatment centers for people with thalassemia and thrombosis.
Health Education and Outreach
Raising overall awareness about hemophilia is critical to advancing public health. To help achieve this, we support education and outreach activities for health promotion and wellness programs. For example, CDC funds the National Hemophilia Foundation's "Do the 5!" campaign. This awareness campaign focuses on five strategies to help people with hemophilia live a longer and healthier life.
CDC currently collaborates with organizations such as the National Hemophilia Foundation and the Hemophilia Federation of America on education and outreach projects that promote wellness for people with hemophilia.
Projects are currently underway to better understand and meet challenges for the following populations:
- Families of persons with a recent hemophilia diagnosis
- Young adults who are transitioning to managing their hemophilia care independently
- People with hemophilia who will benefit from healthy weight programs
- Aging adults with hemophilia
- Other hard-to-reach populations
- Learn about World Hemophilia Day
- Hemophilia Information
- CDC's Division of Blood Disorders
- National Hemophilia Foundation
- Hemophilia Federation of America
- Soucie JM, Nuss R, Evatt, B, Abdelhak A, Cowan L, Hill H, Kolakoski M, Wilber N, and the Hemophilia Surveillance System Project Investigators. Mortality among males with hemophilia: relations with source of medical care. Blood 2000; 96:437-442.
- Soucie JM, Symons J, Evatt B, Bretler D, Huszti H, Linden J, and the Hemophilia Surveillance System Project Investigators. Home-based factor infusion therapy and hospitalization for bleeding complications among males with haemophilia. Haemophilia 2001;7:198-206.
CDC works 24/7 saving lives and protecting people from health threats to have a more secure nation. A US federal agency, CDC helps make the healthy choice the easy choice by putting science and prevention into action. CDC works to help people live longer, healthier and more productive lives.
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