Treatment for hemophilia today is very effective. The missing clotting factor is injected into the bloodstream using a needle. Bleeding stops when enough clotting factor reaches the spot that is bleeding.
Bleeding should be treated as quickly as possible. Quick treatment will help reduce pain and damage to the joints, muscles, and organs. If bleeding is treated quickly, less blood product is needed to stop the bleeding.
With an adequate quantity of treatment products and proper care, people with hemophilia can live perfectly healthy lives. Without treatment, most children with severe hemophilia will die young. An estimated 400,000 people worldwide are living with hemophilia and only 25% receive adequate treatment. The World Federation of Hemophilia is striving to close this gap.
Factor concentrates are the treatment of choice for hemophilia. They can be made from human blood (called plasma-derived products) or manufactured using genetically engineered cells that carry a human factor gene (called recombinant products). Factor concentrates are made in sophisticated manufacturing facilities. All commercially prepared factor concentrates are treated to remove or inactivate blood-borne viruses.
Cryoprecipitate is derived from blood and contains a moderately high concentration of clotting factor VIII (but not IX). It is effective for joint and muscle bleeds, but is less safe from viral contamination than concentrates and is harder to store and administer. Cryoprecipitate can be made at local blood collection facilities.
In fresh frozen plasma (FFP) the red cells have been removed, leaving the blood proteins including clotting factors VIII and IX. It is less effective than cryoprecipitate for the treatment of hemophilia A because the factor VIII is less concentrated. Large volumes of plasma must be transfused, which can lead to a complication called circulatory overload. FFP is still the only product available for treatment of hemophilia A and B in some countries.
People with mild hemophilia A may sometimes use desmopressin (also called DDAVP) to treat minor bleeding. DDAVP is a synthetic hormone that stimulates the release of factor VIII.
New panel discussion: New Developments in Treatment for Bleeding Disorders – Novel Therapies, is a cure for hemophilia around the corner?
While clotting factor concentrates remain, by far, the mainstay of the management of hemophilia, a number of novel therapies under development have the potential to dramatically change the treatment landscape. Leading hematologists and patient advocates from around the world debate the potential of factor mimetics, antithrombin therapies, and gene therapy to change the lives of all people living with bleeding disorders.
Panel: David Lillicrap, Edward Tuddenham, Glenn Pierce, Declan Noone, chaired by David Page.
The opinions expressed in this video are those of the individual panelists and not the official position of the World Federation of Hemophilia.
Updated March 2016