How Is Hemophilia Treated?
Treatment With Replacement Therapy
The main treatment for hemophilia is called
replacement therapygiving or replacing the clotting factor thats
too low or missing. Concentrates of clotting factor VIII (for hemophilia A) or
clotting factor IX (for hemophilia B) are slowly dripped in or injected into a
vein.
Clotting factor concentrates can be made from human
blood that has been treated to prevent the spread of diseases, such as
hepatitis. With the new methods of screening and treating donated blood, the
risk of developing an infectious disease from clotting factors taken from human
blood is now very small.
To further reduce that risk, you or your child can
take clotting factor concentrates that dont use human blood. These are
called recombinant clotting factors. Clotting factors are easy to store, mix,
and use at homeit takes only about 15 minutes to receive the factor.
You may have replacement therapy on a regular basis
to prevent bleeding. This is called preventive or prophylactic
(PRO-fih-lac-tik) therapy. Or, you may only need replacement therapy to stop
bleeding when it occurs. This use of the treatment, on an as-needed basis, is
called demand therapy. Therapy thats given as needed is less intensive
and less expensive than preventive therapy. However, there is a risk that
bleeding will cause damage before the as-needed treatment is given.
Complications of Replacement Therapy
Complications of replacement therapy include:
- Developing antibodies, which are proteins that
act against the clotting factors
- Developing viral infections from human clotting
factors
- Damage to joints, muscles, or other parts of the
body resulting from delays in treatment
Antibodies to the clotting factor.
Antibodies destroy the clotting factor before it has a chance to work. This is
a very serious problem, because it makes the main treatment for
hemophiliareplacing clotting factorsno longer effective.
Antibodies to clotting factor develop in about 20
percent of people with severe hemophilia A and 1 percent of people with
hemophilia B.
When antibodies develop, doctors may use larger
doses of clotting factors or try different sources of the clotting factor.
Sometimes, the antibodies go away. Researchers are studying ways to deal with
antibodies to clotting factors.
Viruses from human blood factors.
The viruses that cause AIDS (HIV) and hepatitis can be carried in clotting
factors. However, there has been no documented case of these viruses being
transmitted during replacement therapy for about a decade. Transmission of
viruses has been prevented by:
- Careful screening of blood donors
- Testing of donated blood products
- Treating donated blood products with a detergent
and heat to destroy viruses
- Vaccinating people with hemophilia for hepatitis
A and B
Researchers continue to find ways to make blood
products safer.
Home Treatment With Replacement
Therapy
Both preventive and as-needed replacement therapy
can be done at home. Many people learn to do the infusions at home for their
child or for themselves. Home treatment has several advantages:
- You or your child can get treatment quicker when
bleeding happens. Early treatment means that fewer complications are likely to
occur.
- Fewer visits to the doctor or emergency room are
needed.
- Home treatment costs less than treatment in a
medical care setting.
- Home treatment helps children accept treatment
and take responsibility for their own health.
Discuss options for home treatment with your doctor
or your childs doctor. A doctor or other health care provider can teach
you the steps and safety procedures for home treatment. Another valuable
resource for learning about home treatment is hemophilia treatment centers
(discussed under Living With
Hemophilia).
Vein access devices can be surgically implanted to
make it easier to get into a vein for treatment with replacement therapy. These
devices can be helpful when such treatment occurs often. However, infections
can be a problem with these devices. Your doctor can help you decide whether
this type of device is right for you or your child.
Other Types of Treatment
Desmopressin
Desmopressin (DDAVP) is a man-made hormone used to
treat people with mild to moderate hemophilia A. DDAVP cant be used to
treat hemophilia B or severe hemophilia A.
DDAVP stimulates the release of stored factor VIII
and von
Willebrand factor and increases the level of these proteins in your blood.
Von Willebrand factor carries and binds factor VIII, which then can stay in the
bloodstream longer.
DDAVP usually is given by injection or in a nasal
spray. Because the effect of this medicine wears off when used often, its
given only in certain situations. For example, your doctor may have you take
this medicine prior to dental work or before playing certain sports to prevent
or reduce bleeding.
Antifibrinolytic Medicines
Antifibrinolytic medicines (including tranexamic
acid and aminocaproic acid) may be used with replacement therapy. They're
usually given as a pill, and they help keep clots from breaking down.
Theyre most often used:
- Before dental work
- For treating bleeding from the mouth or nose
- For mild intestinal bleeding
Gene Therapy
Researchers are trying to develop ways to correct
the defective genes that cause hemophilia to cure the disorder. Such gene
therapy hasnt yet developed to the point that its an accepted
treatment. But researchers continue to test gene therapies for hemophilia in
clinical trials.
Which Treatment Is Best for You?
The type of treatment you or your child receives
depends on several things, including how severe the hemophilia is, what
activities you will be doing, and what dental or medical procedures you will be
having.
- Mild hemophiliaReplacement therapy
isnt usually needed for mild hemophilia. But DDAVP is sometimes given to
raise the bodys levels of factor VIII.
- Moderate hemophiliaYou may need replacement
therapy only when bleeding occurs or to prevent bleeding that could occur when
participating in some activity. DDAVP is another treatment option on occasion,
prior to having a procedure or doing an activity that increases the risk of
bleeding.
- Severe hemophiliaYou usually need
replacement therapy to prevent bleeding that could cause permanent damage to
your joints, muscles, or other parts of the body. Typically, replacement
therapy is given at home two or three times a week. It may be needed on a
long-term basis or just for short periods when you expect to do an activity
that might increase your risk of bleeding. However, some people with severe
hemophilia receive treatment only when bleeding occurs.
For all types of hemophilia, getting treatment
quickly for bleeding to limit damage is important. Learn to recognize signs of
bleeding. Family members also should learn to watch for signs of bleeding in a
child with hemophilia. Children sometimes ignore signs of bleeding because they
want to avoid the discomfort of treatment. |