Prophylaxis and on-demand treatment for hemophilia: How doctors decide
Fact-checked by After a hemophilia diagnosis, people typically work with their care team to develop a tailored treatment plan. This will likely involve considering both preventive (prophylactic) and on-demand hemophilia treatment options.
People with hemophilia are susceptible to excessive or prolonged bleeding episodes because certain blood clotting factors are dysfunctional or missing. Hemophilia treatments, which work in various ways to promote blood clotting, may be given routinely to prevent bleeds and/or as needed to control bleeds that occur.
Doctors consider various factors when deciding between prophylaxis and on-demand treatment, most particularly the type and severity of the person’s hemophilia. Still, people with severe disease should always receive prophylaxis when possible. Treatment decisions in hemophilia are not static, however — the treatment plan may change over time as symptoms evolve or new options become available.
What is prophylaxis in hemophilia?
Prophylaxis involves routinely administering a medication to prevent or reduce the frequency of bleeding episodes. It is generally given on a regular, set schedule, regardless of bleeding events.
The most common prophylaxis treatment for hemophilia is factor replacement therapy, which, as its name suggests, provides patients with a version of the missing or faulty clotting factor via regular infusions given into the bloodstream.
Other prophylactic medications promote blood clotting through mechanisms that don’t involve replacing the missing factor. These can be especially useful for people who develop inhibitors, a type of neutralizing antibody that targets the delivered clotting factor and may cause replacement therapies to be less effective.
What is on-demand treatment?
On-demand, or episodic treatment, involves administering a medication to stop an active bleeding event. It is not given on a set schedule, but rather as needed to control bleeds that occur. Some people only use on-demand therapy. Others are on prophylaxis, but still use on-demand treatments if a breakthrough bleed occurs.
Factor replacement therapies are also used as on-demand treatments, but other medications, called bypassing agents, can also be used on an on-demand basis in people who develop inhibitors.
How doctors decide which approach to use
Choosing between prophylaxis and on-demand treatment is mainly determined by the type and severity of the person’s hemophilia. Treatment guidelines for hemophilia A and hemophilia B — the most common forms of the disease — base the decision on disease severity:
- Severe (clotting factor activity less than 1% of normal): Prophylaxis should be the standard of care when available, and should be started as early in life as possible.
- Moderate (clotting factor activity 1%-5%): Prophylaxis may be considered for patients with frequent or serious bleeding episodes.
- Mild (clotting factor activity 5%-40%): Prophylaxis may be considered for patients with frequent or serious bleeding episodes; on-demand treatment is often used when bleeds are minor and infrequent.
Cost and treatment access may also factor into the decision. On-demand treatment is more commonly used in resource-limited settings. Another factor is lifestyle — people who are more physically active may require prophylaxis to prevent traumatic bleeds.
Hemophilia C, a rarer form of hemophilia, often does not require treatment, though it may be needed when a person’s bleeding risk is elevated, such as during surgery.
Benefits and drawbacks of each strategy
The relative benefits and drawbacks of prophylaxis versus episodic treatment depend on the individual situation.
It is now well-recognized that preventive treatment for hemophilia lowers annual bleeding rates and prevents joint damage more effectively than on-demand treatment. Data consistently show that preventive treatment leads to better long-term health outcomes and a higher quality of life for people with severe disease.
Importantly, though, prophylaxis comes with the burden of needing regular infusions and frequent access to healthcare providers and treatment centers. As such, on-demand treatment may reduce the burden and cost of treatment for patients who experience infrequent, minor bleeding.
Can the treatment strategy change over time?
Once a treatment plan is determined, that doesn’t mean it will stay the same over time. Treatment adjustments may be needed if bleeding patterns change, joint damage occurs, or if a person develops inhibitors. Lifestyle and access to care can also influence these decisions.
For example, people with mild hemophilia who started with on-demand treatment may switch to prophylaxis if they start having more frequent or serious bleeds. Individuals using on-demand treatment may be able to switch to prophylaxis if new options become available in their region.
Patients should have regular discussions with their care team about how well their treatment is working and whether any changes are needed.
Questions to ask about your treatment plan
After a hemophilia diagnosis, a few questions to ask a care provider include:
- How severe is my hemophilia, and what is my bleeding risk?
- Do you recommend prophylaxis or on-demand therapy?
- What are the pros and cons of each of my treatment options?
- How do I know when to treat a bleed with on-demand therapy?
- What are some red flags to look for that might mean my treatment needs to be changed?
Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
