Von Willebrand disease is a lifelong bleeding condition that affects how well blood clots, causing people to bleed more than expected from injuries, surgeries, or even minor cuts. While there is no cure, a range of treatments and lifestyle strategies can help manage symptoms and allow most people to live full, active lives.

Helping Blood Clot When It Doesn’t Work as It Should

When someone has Von Willebrand disease, the main goal of treatment is to help their blood clot properly during bleeding episodes or prevent bleeding from happening in situations that might cause it. This is important because the disease creates a double problem: the body either doesn’t have enough of a protein called von Willebrand factor, or the protein doesn’t work correctly. On top of that, another clotting protein called factor VIII may also be lower than it should be because von Willebrand factor normally protects it.^[1][12]

The treatment approach depends heavily on which type of Von Willebrand disease a person has and how severe their symptoms are. Some people have such mild symptoms that they may not even know they have the condition until they have surgery or dental work and notice they bleed longer than expected. Others may have more frequent and serious bleeding that requires regular medical attention.^[1][3]

Treatment choices also depend on where the bleeding is happening, how severe it is, and what activities or procedures might trigger bleeding. For example, someone might need treatment before a planned surgery, during a heavy menstrual period, or after an unexpected injury. Medical societies and expert groups have developed guidelines that help doctors choose the right treatment for each situation.^[9][12]

Beyond the standard treatments that have been used for years, researchers are also working on new therapies through clinical trials. These studies explore innovative approaches that might offer better results or fewer side effects for people living with this condition.^[13]

Established Treatments That Help Control Bleeding

The first-line treatment for many people with Von Willebrand disease, particularly those with type 1 and certain forms of type 2, is a medication called desmopressin, also known by the brand name DDAVP. This is a synthetic version of a natural hormone that tells the body to release von Willebrand factor that is stored in the cells lining blood vessels. Essentially, it helps the body use its own reserves of the clotting protein.^[9][12]

Desmopressin can be given in several ways. Some people use a nasal spray that they spray into their nose, where the medicine is absorbed into the bloodstream. Others receive it as an injection under the skin or through a vein. The nasal spray form is particularly convenient because people can use it at home when they need it, such as before dental work or during a nosebleed that won’t stop.^[9][14]

However, desmopressin doesn’t work for everyone. Before using it regularly, doctors typically perform a test dose when the person is not bleeding to see how their body responds. They measure the von Willebrand factor levels in the blood after giving the medication to check if the levels rise enough to be helpful. If the medication works well, the person is considered a “responder” and can use it when needed.^[12][16]

Common side effects of desmopressin include headaches, a temporary increase in heart rate, and low blood pressure. A more serious concern is that repeated doses can cause the sodium levels in the blood to drop too low, which in rare cases can lead to seizures. Because of this risk, doctors may be cautious about using desmopressin in young children under five years old and in elderly people.^[14][16]

For people who don’t respond to desmopressin or who have more severe forms of the disease, doctors turn to von Willebrand factor replacement therapy. This involves giving concentrated von Willebrand factor directly into the bloodstream through a vein. These concentrates often also contain factor VIII, since both proteins are needed for proper clotting.^[9][13]

Several brands of von Willebrand factor concentrates are available in the United States. Most of these are made from human blood plasma that has been carefully processed to remove viruses and other infectious agents. The brands include Alphanate SD, Humate P, Koate DVI, and Wilate. These products contain both von Willebrand factor and factor VIII in varying ratios.^[16]

The doses and frequency of these concentrates depend on what they’re being used for. For someone having major surgery, treatment might start before the operation and continue for seven to fourteen days afterward to keep bleeding risks low. For minor procedures, treatment might only be needed for three to five days. The goal is to maintain sufficient levels of both von Willebrand factor and factor VIII until the risk of bleeding has passed.^[13]

Another helpful medication category is antifibrinolytic drugs, which include tranexamic acid and aminocaproic acid. These medicines work differently from desmopressin or factor concentrates. Instead of helping blood clot, they help stabilize clots that have already formed, preventing them from breaking down too quickly. They are particularly useful for mild bleeding from mucous membranes, such as nosebleeds, bleeding gums, or heavy menstrual periods.^[9][13]

Tranexamic acid and aminocaproic acid can be taken as pills or given through a vein. For menstrual bleeding, women often take pills during their periods to reduce the amount of blood loss. For dental procedures, these medications might be used along with other treatments to prevent bleeding from the gums.^[5]

In emergency situations where other treatments are not available, doctors might use cryoprecipitate, which is a blood product that contains von Willebrand factor along with other clotting proteins. However, because cryoprecipitate is not treated to kill viruses as thoroughly as factor concentrates, it carries a higher risk of transmitting infections. For this reason, it is generally avoided unless no other options exist.^[16]

Sometimes, for people with severe bleeding that doesn’t respond to other treatments, doctors may consider platelet transfusions. Platelets are blood cells that help form clots, and transfusing them can provide an extra boost to the clotting system. This is most often considered for people with type 3 Von Willebrand disease who have very severe bleeding.^[13]

There are also topical treatments that can be applied directly to bleeding wounds. Fibrin sealants are glue-like substances that can be placed on surface wounds or used during dental surgery to help stop bleeding. While they may be helpful in certain situations, their safety and effectiveness are still being evaluated.^[13]

Innovative Approaches Being Tested in Research

A major advancement in the treatment of Von Willebrand disease came with the approval of recombinant von Willebrand factor, marketed under the brand name Vonvendi. This was the first von Willebrand factor product made in a laboratory without using human blood or plasma, which virtually eliminates the risk of transmitting infections that can be present in blood-derived products.^[15]

Vonvendi was initially approved by the United States Food and Drug Administration in 2015 based on a Phase III clinical trial. In this trial, 22 patients with Von Willebrand disease used Vonvendi to treat 192 bleeding episodes. The results showed that the treatment controlled bleeding effectively, with doctors rating the results as good or excellent in managing minor, moderate, and major bleeds. In fact, excellent control was achieved in about 97 percent of cases, and a single infusion was enough to stop the bleeding in about 82 percent of episodes.^[13][15]

The mechanism of action of Vonvendi is straightforward: when infused into the bloodstream, it acts like the body’s natural von Willebrand factor. It helps platelets stick together and form clots, and it also protects factor VIII from being broken down too quickly. Because it’s made through recombinant technology using cells grown in the laboratory, it doesn’t carry the same risks as products made from pooled human blood.^[15]

Over time, the approved uses for Vonvendi have expanded. In 2018, it received approval for use before and after surgery to manage bleeding in adults. Then in 2022, it was approved for regular preventive treatment, called prophylaxis, in adults with severe type 3 Von Willebrand disease who had previously been using on-demand therapy. Most recently, in 2025, the approval was expanded even further to include routine prophylaxis for adults with any type of Von Willebrand disease, and to include both adults and children for on-demand treatment of bleeding episodes and management of bleeding during surgery.^[13][15]

Vonvendi can be used alone or in combination with recombinant factor VIII, depending on what the doctor recommends. For emergency surgery, both von Willebrand factor and factor VIII are needed immediately to ensure proper clotting. However, for planned elective procedures, giving Vonvendi early can help stabilize the body’s own factor VIII levels.^[13]

Clinical trials have shown that Vonvendi has a positive safety profile. The most common side effects reported include headaches, nausea, and dizziness. However, because it is a protein product, there is always a potential for allergic reactions. People who are allergic to mice or hamsters should not use Vonvendi because the cells used to make it come from these animals.^[15]

The ability to use Vonvendi as prophylaxis represents a significant shift in treatment strategy. Instead of only treating bleeds after they happen, people with severe type 3 Von Willebrand disease can now receive regular infusions to reduce the frequency of bleeding episodes. This approach is similar to what has been used for years in hemophilia care, where regular infusions of clotting factors have dramatically improved quality of life.^[15]

Clinical trials for Von Willebrand disease treatments are conducted in phases. Phase I trials focus on safety, testing a new treatment in a small group of people to evaluate side effects and determine safe dosing ranges. Phase II trials expand the group and begin to assess how well the treatment works, measuring whether it actually improves symptoms or laboratory values. Phase III trials involve larger groups of patients and often compare the new treatment to existing standard treatments or a placebo.^[13]

Eligibility for clinical trials varies depending on the study. Researchers typically look for people with specific types of Von Willebrand disease, certain age ranges, and particular bleeding patterns. Clinical trials for Von Willebrand disease have been conducted in many locations, including the United States, Europe, and other regions. People interested in participating can search for trials through healthcare providers or clinical trial registries.^[13]

Research continues into other innovative approaches as well. Scientists are exploring ways to improve the effectiveness of existing treatments, develop longer-lasting products that require less frequent infusions, and find new molecules that might work through different mechanisms. Some research also focuses on understanding why certain people respond better to specific treatments, which could eventually lead to more personalized treatment strategies.^[12]

Most common treatment methods

• Desmopressin (DDAVP)
  • A synthetic hormone that stimulates release of von Willebrand factor stored in blood vessel cells
  • Available as nasal spray, injection under the skin, or infusion through a vein
  • First-line treatment for type 1 and some type 2 Von Willebrand disease patients who respond to it
  • Requires a test dose to determine if a person will respond effectively
  • Side effects include headaches, low blood pressure, temporary increase in heart rate, and potential for low sodium levels
• Von Willebrand Factor Concentrates
  • Plasma-derived products made from human blood that has been processed to remove viruses
  • Brands available in the United States include Alphanate SD, Humate P, Koate DVI, and Wilate
  • Contain both von Willebrand factor and factor VIII
  • Used for people who don’t respond to desmopressin or have severe disease
  • Given through a vein for treatment of bleeding episodes and prevention during surgery
• Recombinant Von Willebrand Factor (Vonvendi)
  • First laboratory-made von Willebrand factor without using human blood or plasma
  • Approved for on-demand treatment of bleeding episodes in adults and children
  • Approved for perioperative bleeding management during and after surgery
  • Approved for routine prophylaxis to reduce bleeding frequency in adults with severe type 3 and other types
  • Can be used alone or with recombinant factor VIII
• Antifibrinolytic Medications
  • Include tranexamic acid and aminocaproic acid
  • Help stabilize blood clots after they form and prevent them from breaking down
  • Particularly useful for mild mucous membrane bleeding such as nosebleeds and heavy menstrual periods
  • Can be taken orally as tablets or given through a vein
  • Often used as additional therapy alongside other treatments
• Supportive Treatments
  • Topical fibrin sealants applied directly to surface wounds or used during dental surgery
  • Platelet transfusions for severe bleeding unresponsive to other therapies, particularly in type 3 disease
  • Cryoprecipitate as emergency treatment when other options are unavailable, though carries higher infection risk