Rare Disorders Rare Disorders
Centers for Disease Control and Prevention
At a comprehensive treatment center, a team of experienced healthcare professionals can work together to treat people with rare or complex medical conditions such as hemophilia.
Comprehensive treatment centers are specialized healthcare centers that bring together a team of doctors, nurses, and other health professionals experienced in treating people with rare or complex chronic medical conditions.1
CDC’s Division of Blood Disorders, along with other federal agencies, supports a network of 135 comprehensive treatment centers able to meet the unique challenges of people with hemophilia. Hemophilia is an inherited blood disorder that can lead to chronic health problems such as joint disease over time. However, if the disorder is managed properly, people with hemophilia can live long, healthy lives.
A CDC study of 3,000 people with hemophilia showed that those who used 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.2 Similarly, people who used a treatment center were 40% less likely to be hospitalized for bleeding complications.3
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. For example, the National Hemophilia Foundation partners with hemophilia comprehensive treatment centers and CDC to educate people with this disorder about the top five things they can do to reduce complications. (For more information about how to help prevent complications, visit www.hemophilia.org*.
Advancing Research and Ensuring Safety
One of the major challenges facing researchers and scientists who work on rare disorders such as hemophilia is lack of access to uniform data. Hemophilia treatment centers can participate in CDC’s Universal Data Collection (UDC) program, which collects a consistent set of data to monitor the health of people with hemophilia. The data are used to advance research for this condition and other related disorders.
Another important aspect of the UDC program is ensuring that blood products used by people with hemophilia do not contain known viruses. Participants in the UDC program give a blood sample each year during their annual clinic visit. A portion of this sample is tested for certain viruses. This testing can be an early indication if certain harmful viruses are being transmitted through the Nation’s blood products. Today, blood products in the U.S. blood supply are considered safe from known viruses. However, continued testing through the UDC program can help to ensure safety – both for the hemophilia community and others using blood products.4
CDC also supports treatment and research center networks for other bleeding and clotting disorders. There are now six treatment centers for people with thalassemia and eight for people with thrombosis.5 In addition, CDC would like to conduct research to identify which factors make the comprehensive treatment center model a success. This information could be used to help treat and control other rare and complex medical conditions.