Hemophilia is a complex disease that requires care from medical providers who are experienced in treating it.
The symptoms of hemophilia — bleeding and bruising — are caused by having a low level of clotting factors in the blood. Therefore, people with hemophilia need to replace those factors so that their blood is able to clot appropriately.
There are multiple treatments, medications, and other supportive therapies that can mitigate the symptoms of hemophilia A.
Medication
The main focus of hemophilia treatment is to replace clotting factors in the blood. There are two basic types of clotting factor replacements:
Plasma-derived factor concentrates, derived from donated human plasma
Recombinant factor concentrates developed from culturing cells
Both of these treatments are given by injection under a person’s skin or directly into a vein.
People can receive these concentrated treatments for bleeding that has already occurred, or to prevent future bleeding. Receiving them to prevent future bleeding incidents is called prophylactic care because it is preventive. Providing treatment to stop bleeding that is already occurring is called episodic treatment.
People can administer clotting factor replacements to themselves or have a family member or other caregiver administer them.
Plasma-Derived Factor Concentrates
Plasma-derived factor concentrates are blood clotting factors that come from plasma donated by other people.
Plasma is the liquid part of blood, which contains many proteins, including antibodies and clotting factors. It is first separated from the red blood cells, then it goes through additional processing to break it down into its different components, including clotting factors. These isolated clotting factors can then be freeze-dried for use in therapeutic treatments.
Plasma and its components are tested for bacteria and viruses. The blood product is further treated to kill any pathogens (disease-causing microorganisms) that might still be present.
These drugs include:
Antihemophilic factor (Hemofil-M)
Antihemophilic factor and von Willebrand factor complex (Alphanate)
Recombinant Factor Concentrates
Recombinant factor concentrates are developed by culturing cells and then manipulating the DNA in those cells to isolate the blood factor needed. This method allows scientists to create a key factor used to treat hemophilia A called factor 8 concentrate. This factor can be created without plasma or albumin, another protein present in blood plasma. As a result, it’s unlikely the product will contain any viruses.
The injection drug emicizumab-kxwh (Hemlibra) functions by working in the place of natural blood clotting factors. It is used to prevent bleeding episodes or to lessen their occurance.
Antifibrinolytics
Antifibrinolytics are clot-promoting drugs that prevent the breakdown of a protein called fibrin.
Desmopressin acetate (DDAVP) is a synthetic chemical that is administered by a nasal spray. It resembles a hormone made by the pituitary gland called arginine vasopressin.
DDAVP causes the body to produce more factor 8. People with mild hemophilia A can use DDAVP, but it is less useful for those with more severe disease.
Epsilon aminocaproic acid (Amicar) is given intravenously or orally in pill or liquid form to stop blood clots from breaking down.
It is not given on a regular basis, but it has been used to help control bleeding when a person with hemophilia A undergoes a dental procedure.
Fibrin Sealants
Medicines called fibrin sealants can be put directly on a wound to help the blood clot.
Minor cuts or bleeding from procedures, such as dental procedures, should also receive basic first aid care, but these sealants can assist with healing.
Gene Therapy
In 2023, the U.S. Food and Drug Administration (FDA) approved the first gene therapy for severe hemophilia A. Known as valoctocogene roxaparvovec-rvox (Roctavian), this gene therapy is only approved for adults. It is a onetime gene therapy administered by intravenous infusion. It has a viral vector that contains a gene for the clotting factor 8.
Bypassing Agents
Some people with hemophilia can have inhibitors that prevent clotting factor concentrates from working. The body may identify this factor as a foreign substance and try to destroy it.
In these cases, a physician may use a bypass agent to help the patient with clotting.
These agents do not replace the missing factors. Instead they help the body form a normal clot by bypassing the factors that are blocked by these inhibitors.
In October 2024, the FDA approved the new drug marstacimab-hncq (Hympavzi). It is the first non-factor treatment, and it works by reducing the amount of a naturally occurring anticoagulation protein, called tissue factor pathway inhibitor.
As a result, the body generates an enzyme critical in blood clotting called thrombin. The drug is approved for people over age 12 with hemophilia A without factor 8 inhibitors or hemophilia B without factor 9 inhibitors (neutralizing antibodies).
Delivery Devices to Reduce Injections
Since hemophilia treatment can involve so many injections, some people opt to use a central venous access device (CVAD).
These are medical devices implanted under a person’s skin to provide frequent injections. The most common type is a port-a-cath, but other types of CVADs exist.
Port-a-caths remove the need for a clinician to repeatedly find a vein for injections, so they are particularly helpful in children. But the risks of port-a-caths include infection and blood clots, including thrombosis (a large blood clot).
Rehabilitation and Therapy
People with hemophilia may experience pain in their joints from internal bleeding. Sometimes the joints can become permanently damaged due to the bleeding, which is called hemophilic arthropathy.
Physical therapy can help strengthen a person’s muscles to reduce this pain and help preserve joint function. A physical therapist has many options for helping to preserve joint function, including manual therapy, corrective exercises, orthotics, braces, or splinting. They can also use transcutaneous electrical nerve stimulation, where a low-voltage electrical current is passed through the skin to stimulate the nerves.
Complementary and Integrative Approaches
For some people, complementary therapies can help with the pain and stress of dealing with hemophilia A. These therapies can include acupressure, acupuncture, yoga, and reflexology, among others.
Pain Management
People with pain due to hemophilia A have multiple options for pain management. They can try the PRICE method, an acronym for Protect, Rest, Ice, Compression, and Elevation, to help treat an injury.
They can also often take certain over-the-counter pain relievers, such as acetaminophen (Tylenol), but they should avoid ibuprofen (Advil) and aspirin, due to bleeding risks. They may also be able to take non-opioid pain medications such as a COX-inhibitor (Celebrex).
Mental Health Treatment
Dealing with a diagnosis of hemophilia A can impact emotional health, and many people with a chronic illness also have a mental health disorder such as depression or anxiety.
