Unravel the enigma of hemophilia, a rare genetic disorder that challenges blood clotting. Discover treatments that empower individuals to manage their condition and lead active lives.
What is Hemophilia?
Hemophilia is a rare, inherited bleeding disorder in which the blood does not clot properly. It is caused by a deficiency or abnormality in specific clotting proteins, known as clotting factors, in the blood. When a person with hemophilia experiences bleeding, it can be difficult to stop and may continue for a prolonged period. Hemophilia primarily affects males, although females can also be carriers of the condition.1
There are two main types of hemophilia: hemophilia A and hemophilia B. Hemophilia A is the most common type and is caused by a deficiency or abnormality in clotting factor VIII. Hemophilia B, also known as Christmas disease, is caused by a deficiency or abnormality in clotting factor IX.
The severity of hemophilia can vary from mild to severe, depending on the level of clotting factor deficiency. Individuals with mild hemophilia may experience occasional bleeding episodes, while those with severe hemophilia may experience frequent and prolonged bleeding.
How Does Hemophilia Impact Daily Life?
Hemophilia can significantly impact the daily life of affected individuals. People with hemophilia need to take extra precautions to prevent bleeding episodes and manage their condition effectively. This may involve:
- Avoiding activities that carry a high risk of injury or bleeding
- Regular prophylactic infusions of clotting factor concentrates to prevent spontaneous bleeding
- Seeking prompt medical attention for any bleeding episodes
- Undergoing physiotherapy to maintain joint mobility and prevent muscle atrophy
- Managing pain and discomfort associated with bleeding episodes
Individuals with hemophilia may also face challenges in areas such as education, employment, and social activities due to the limitations imposed by their condition. However, with proper medical care and support, people with hemophilia can lead full and active lives.
Treatment Options for Hemophilia
The primary treatment for hemophilia is clotting factor replacement therapy. This involves infusing the missing or deficient clotting factor into the bloodstream to help control bleeding and prevent further episodes. Clotting factor concentrates can be administered intravenously (IV) or subcutaneously (under the skin).2
Other treatment options for hemophilia include:
- Desmopressin (DDAVP): A synthetic hormone that helps increase the levels of clotting factor VIII in the blood, reducing the risk of bleeding.
- Emicizumab: A monoclonal antibody that helps prevent and control bleeding in individuals with hemophilia A.
- Gene therapy: An experimental treatment that aims to correct the genetic defect responsible for hemophilia by introducing a functional clotting factor gene into the body.
The choice of treatment depends on the type and severity of hemophilia, as well as the individual’s specific needs and preferences. Regular monitoring and follow-up care are essential to ensure effective management of hemophilia and prevent complications.
Learn More About Hemophilia
For more information and resources on hemophilia, individuals can refer to the following sources:
- World Federation of Hemophilia (WFH): A global organization dedicated to improving the lives of people with hemophilia and related bleeding disorders.
- National Hemophilia Foundation (NHF): A US-based organization that provides support, education, and advocacy for individuals with hemophilia and their families.
- Hemophilia Federation of America (HFA): A US-based organization that works to improve the lives of people with hemophilia and related bleeding disorders through advocacy, education, and research.