Bleeding disorders such as hemophilia and von Willebrand Disease are inherited problems in coagulation (blood clotting) caused by missing or poorly functioning proteins in the blood (clotting factors.)
The two most common types of hemophilia are Factor VIII and Factor IX Deficiency. Factor VIII Deficiency, also called classic hemophilia or hemophilia A, occurs in about 1 in every 10,000 live male births in the United States. Factor IX Deficiency or hemophilia B, also called Christmas Disease or hemophilia B, is more rare. Because hemophilia is carried on the X chromosome (one in the pair that determine sex), most patients with hemophilia are males.
Von Willebrand Disease is the most common of all inherited bleeding disorders, affecting up to one in every 100 individuals. It is caused by two missing or poorly functioning clotting factors: von Willebrand Factor and Factor VIII. Von Willebrand Factor is a "glue-like" adhesive protein that carries and protects Factor VIII in the blood and sticks to blood vessel walls when they are injured. Von Willebrand Disease is inherited equally among men and women.
Additionally, other, very rare factor deficiencies also cause clotting problems similar to classic hemophilia or von Willebrand Disease. Most of these deficiencies affect men and women equally.
Several centers in the United States are now studying gene therapy to cure certain inherited bleeding disorders. Until a cure is readily available, replacing the missing or poorly functioning clotting factor is the treatment for severe and moderate hemophilia and certain types of von Willebrand Disease. Most persons with severe or moderate bleeding disorders are able to prevent or treat bleeding through intravenous home infusion of clotting factors. Some mild inherited bleeding disorders may be treated with medications such as desmopressin (or Stimate ) or Amicar (aminocaprioc acid).
Comprehensive care is coordinated health care for persons with inherited bleeding disorders through a family-centered approach by a multidisciplinary team. The team includes specially trained physicians, nurses, social workers, physical therapists, orthopedic surgeons, infectious disease specialists, dentists, genetic counselors, nutritionists, and educational consultants. Comprehensive care focuses on the physical, emotional, social, educational, financial and workplace impact of bleeding disorders on the person and family. The comprehensive care team collaborates closely with the patient's medical home (primary care provider) within their community.
Studies have shown that patients with inherited bleeding disorders who have their care coordinated through a comprehensive bleeding disorder center live longer and healthier lives than those who receive care from a hematologist alone.
The CSHCN Hemophilia Program is a legislatively-enacted program through the Virginia Department of Health, Division of Child and Adolescent Health for the care and treatment of persons with hemophilia and other inherited bleeding disorders. Virginia recognizes that the ongoing medical costs of treating such bleeding disorders often exceed the financial capacity of families, despite insurance coverage. The program supports a system of coordinated, family-oriented, multidisciplinary services for persons of all ages with bleeding disorders