Clotting factor replacement therapy has been the mainstay of haemophilia treatment for decades, making a problem that used to be life-threatening much easier to deal with. But as it gets easier to get care, a new problem is coming up: the risk of overmedicating haemophilia.
Clotting factor treatment must be used at the right time to stop excessive bleeding, but using it too much or when it's not needed can cause problems. Lots of people, including doctors, think that "more is better," but using too many clotting factors can cause health problems that you might not expect.
This piece talks about the growing worry about haemophilia patients taking too many medicines, the problems that come with long-term clotting factor replacement, and why we need a more balanced approach.
Clotting Factor Therapy
People with haemophilia have an inherited disorder that makes them unable to make enough of two clotting factors, Factor VIII (Haemophilia A) or Factor IX (Haemophilia B).
These factors help the blood clot properly. People who have haemophilia bleed for a long time after even small accidents. They also have "spontaneous internal bleeds," which happen inside the body, mostly in the joints and muscles.
Clotting factor therapy, which is given through infusions, replaces the proteins that are missing. This stops severe bleeding events and lets patients live mostly normal lives. Balance is important, though, as it is with all medical treatments.
Risks of Using Clotting Factor Therapy Too Much
While factor replacement therapy is important for managing haemophilia, using it too much—either by giving unnecessary doses, treating small bleeds too harshly, or giving too many prophylactic infusions—can cause major problems:
1. Development of Inhibitors
When people with haemophilia take too many medicines, one of the worst things that can happen is that inhibitors, which are antibodies that stop clotting factor from working, can form.
This makes the treatment useless. Up to 30% of people with serious haemophilia A develop inhibitors. This is usually because they get too many clotting factor infusions.
Inhibitors make treatment much harder and more expensive because they need to be replaced with other methods, like skipping agents, which may not stop bleeding as well.
2. Higher Risk of Thrombosis (blood clots in places they shouldn't be)
People with haemophilia tend to bleed a lot, but too much clotting factor therapy can tip the scales in the opposite direction, making it more likely that blood will clot in a way that isn't normal (thrombosis). It's especially important for older people with haemophilia who may already have heart problems to think about this.
3. Damage to Joints from "Microclots"
In a strange way, clotting factor treatment stops bleeding into joints (hemarthrosis), but too much of it may lead to the formation of microclots, which can hurt joints and last for a long time. This is becoming more of a worry as more haemophilia patients live longer and have problems with their joints.
4: Financial and Mental Toll it Takes
When clotting factor treatment is used too much, it costs healthcare systems, insurance companies, and patients a lot of money. These treatments are among the most expensive in modern medicine, and giving fluids that aren't needed can waste money and hurt the patient's health.
Also, the mental effects of taking too many medications should not be ignored. Patients may become dependent on infusions and feel unsafe or worried without regular doses, even if they aren't needed.
Is Less Really More in Haemophilia Care?
Managing haemophilia isn't just about stopping small bleeding; it's also about making sure the person stays healthy and has a good quality of life. So how can we avoid the risks of giving too many medicines while still giving the best care?
1. Individualized Prophylaxis Plans: Every patient is different and needs a different treatment plan. Personalized prophylaxis, in which the amount of clotting factor given depends on the person's risk of bleeding, level of physical exercise, and way of life, can help stop needless infusions while still protecting against bleeds well.
2. Looking into therapies that don't depend on a factor: Some newer treatments, like monoclonal antibodies can be used instead of standard factor replacement therapy. These treatments lower the chance of developing inhibitors and thrombosis and also cut down on the number of times that patients need to receive infusions.
3. Teaching Patients and Caregivers About Haemophilia Treatment: Patients with haemophilia and their families need to be taught about treatment so they don't overreact to small injuries. Not every bruise or small cut needs clotting factor treatment right away. Knowing the difference between real emergencies and mild bleeds can help you avoid giving the wrong dose.
4. Making progress in personalized medicine: Precision medicine is the way of the future for treating haemophilia. Genetic profiling and new diagnostic tools can help doctors figure out exactly how much treatment a patient really needs, lowering the risks of both under- and over-treatment.
Haemophilia care has come a long way, but now the focus is on improving long-term health instead of just stopping bleeding. People who take too much medicine, often out of fear, can create new risks that are just as dangerous as under-treatment.
We can make sure that people with haemophilia get just the right amount of therapy—no more, no less—by taking a smarter, more personalized approach. This will lead to better results, fewer complications, and a healthier future.
Disclaimer: The views expressed in this article are of the author and not of Health Dialogues. The Editorial/Content team of Health Dialogues has not contributed to the writing/editing/packaging of this article.