Treatment of von Willebrand disease

/Treatment of von Willebrand disease
Treatment of von Willebrand disease2018-05-11T14:52:53+00:00

No. Minor bleeding episodes associated with VWD often do not require medical treatment. For example:

  • Small bruises usually disappear on their own
  • Larger bruises or hematomas can often be controlled by applying cold (an ice pack wrapped in a towel) and elevating the limb
  • Bleeding from minor cuts can be stopped by applying pressure.
  • Nosebleeds may be stopped with simple first aid techniques (See Nosebleeds)

However, sometimes medical treatment is necessary. The type of treatment depends in part on the type of VWD a person has.

Few doctors are familiar with VWD. Even hematologists, who deal with diseases of the blood, are rarely experts in diagnosing and treating bleeding disorders. Many obstetricians and gynaecologists remain unaware of the consequences of VWD for women.

Therefore, the best place for a person with VWD to get treatment is a bleeding disorder treatment centre. (See Comprehensive care)

Once a proper diagnosis has been made, and a treatment plan has been organized, the doctors at the bleeding disorder treatment centre can work with the individual and his/her family physician to provide care.

DESMOPRESSIN

Desmopressin is a synthetic drug which is a copy of a natural hormone. It acts by releasing VWF stored in the lining of the blood vessels. Desmopressin is not made from blood.

Desmopressin is the preferred treatment for Type 1 VWD. It can be taken in three different ways.

  • It can be injected into a vein. Most often, the brand name for this kind of desmopressin is DDAVP, Octostim or Stimate.
  • It can be injected subcutaneously (under the skin). The brand name for this kind of desmopressin is also DDAVP, Octostim or Stimate.
  • It can be taken by nasal spray. The brand name of the nasal spray is often Octostim Spray or Stimate Spray.

Desmopressin is effective for many people with Type 1 VWD. However, different people respond to desmopressin in different ways. Therefore, a doctor needs to do tests to find out each individual’s response to the drug. Ideally, these tests are done before any urgent need for the drug, such as surgery.

Since desmopressin acts by releasing VWF stored in the body, a person cannot take it repeatedly over a short period. A sufficient amount of time, usually 24 hours, must elapse between doses of desmopressin to allow the body to rebuild its stores.

In major surgery, desmopressin alone may not be enough to control bleeding. In such a case, a person should also receive a concentrate of VWF and factor VIII. (See Factor VIII/von Willebrand factor concentrate)

Desmopressin can sometimes have some mild side effects. These are:

  • facial flushing
  • mild headache
  • nausea and abdominal cramps.

Desmopressin is an antidiuretic; that is, it can make the body retain water. Therefore, doctors recommend that after receiving desmopressin people drink only enough fluid to satisfy thirst.

In very rare cases, desmopressin can cause more serious side effects. If a person has a very bad headache or has not been able to urinate 24 hours after taking desmopressin, he/she should go to the bleeding disorder treatment centre or emergency room for help. In infants, fluid overload caused by desmopressin can cause seizures. In the elderly and in people with cardiovascular disease, desmopressin can cause more serious side effects and may not be recommended.

 

CYKLOKAPRON AND AMICAR

(Antifibrinolytic agents)

Cyklokapron (tranexamic acid) and Amicar (aminocaproic acid) are drugs that help to hold a clot in place once it has formed. They act by stopping the activity of an enzyme, called plasmin, which dissolves blood clots.

They do not help to actually form a clot. This means they can not be used instead of desmopressin or factor VIII/VWF concentrate in cases of serious bleeding.

They can be used to hold a clot in place in mucous membranes such as:

  • the inside of the mouth
  • the inside of the nose
  • inside the intestines (the gut)
  • inside the uterus (the womb).

Cyklokapron and Amicar have proven very useful for people with VWD. They are used:

  • before dental work
  • when a person has mouth, nose and minor intestinal bleeding
  • for women with heavy, prolonged menstrual bleeding.

Cyklokapron comes in tablet form. Amicar comes in tablet, liquid and injectable form. They can sometimes have some mild side effects. These are:

  • feeling sick to the stomach (nausea)
  • feeling tired or sleepy
  • feeling dizzy
  • having loose bowel movements (diarrhea)
  • having pain in the stomach.

These mild side effects go away when:

  • a person stops taking the drugs
  • the doctor reduces the dosage.

A person with urinary tract bleeding (blood in the urine) should not take these drugs.

 

FIBRIN GLUE

In the coagulation process, the final clot is made up of fibrin. A product known as fibrin glue is removed from blood and manufactured as a natural clotting agent. It can be applied directly to the site of bleeding. It is especially useful in tooth extractions and surgery. Over a period of 2 to 4 weeks, as healing progresses, the fibrin is aborbed by the body.

FACTOR VIII/VON WILLEBRAND FACTOR CONCENTRATE

Factor VIII/von Willebrand factor concentrate (FVIII/VWF concentrate) is the preferred treatment for:

  • Type 3 VWD
  • most forms of Type 2 VWD
  • for serious bleeding or major surgery in all types of VWD.

This concentrate replaces the missing VWF in the blood long enough to allow clotting to take place.

FVIII/VWF concentrate is made from pooled human plasma. Plasma is a pale yellow liquid contained in blood. The plasma has been fractionated to extract the factor VIII and von Willebrand factor. This means that the different parts of the blood have been separated from each other so that each person can receive only that part which he/she needs.

The plasma used for FVIII/VWF concentrate is screened for blood-borne viruses such as HIV and hepatitis B and C. Any plasma found to contain these viruses is not used. Products made from the remaining plasma are then treated to destroy any viruses that may still be present. Factor concentrates used today have an excellent safety record. However, Parvovirus B19 may still be transmitted.

Humate P is the brand name most commonly used in Canada. Wilate is another concentrate of FVIII/VWF that is available for the treatment of VWD in Canada.

FVIII/VWF concentrate is injected into a vein. It can be administered at a clinic, doctor’s office or emergency room. Many people learn to inject it themselves at home. (See What is home care?)

Other factor VIII concentrates such as monoclonal factor VIII and recombinant factor VIII contain no von Willebrand factor. Therefore, they are of no value in treating VWD.

 

CYKLOKAPRON AND AMICAR

Bleeding in mucous membranes can sometimes be treated with Fibrin, Cyklokapron and Amicar. (See above the treatments for Type 1 VWD.)

 

DESMOPRESSIN

Desmopressin is not often recommended for Type 2 VWD. In most forms of Type 2 VWD, the von Willebrand factor does not work properly. As desmopressin works by releasing stored-up von Willebrand factor, it would only succeed in releasing a larger amount of a protein that does not work.

With Type 2B VWD, desmopressin could make the clumping together of platelets even worse than it already is. This would lower the platelet count or even cause unwanted clots in the bloodstream. Formation of an effective clot would be even more difficult.

However, some individuals with Type 2A VWD have a good response to desmopressin. Therefore, it is recommended that, at the time of diagnosis, tests be done to measure a person’s response to desmopressin.

Nor is desmopressin recommended in the treatment of Type 3 VWD. With this type of VWD, very little VWF is stored in the blood vessel linings so desmopressin would not be effective in releasing stored-up VWF.

Medical options for people with von Willebrand disease

DISORDER PREFERRED TREATMENT ALTERNATIVE
Type 1 Desmopressin FVIII/VWF concentrate
Type 2A Desmopressin, if individual responds FVIII/VWF concentrate
Type 2B FVIII/VWF concentrate  
Type 3 FVIII/VWF concentrate  
N.B. Oral contraceptives, other hormone therapy and antifibrinolytic drugs may also be useful for women with menorrhagia, in particular circumstances.

 

Cryoprecipitate is a blood component made from plasma, containing VWF and factor VIII. It was commonly used to treat VWD in the past. However, because there is no method to kill viruses in cryoprecipitate, it is no longer recommended.

Currently, techniques are being developed to treat cryoprecipitate for viruses. When they are proven effective, cryoprecipitate may once again be used as a treatment for VWD.

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