When your hemophilia treatment stops working: What to do next
Fact-checked by The standard for treating hemophilia A and B is clotting factor replacement therapy, but the immune system sometimes recognizes the supplied clotting factor as a threat and produces antibodies, called inhibitors, that block its activity. This can render clotting factor therapy ineffective. Though hemophilia inhibitors can develop at any time, they typically surface within the first 50 treatment days.
If you have hemophilia and an inhibitor develops and begins compromising your treatment, know that other options are available to manage your hemophilia.
Signs your treatment may not be effective
Inconsistent or missed treatments that result in lower clotting factor activity may increase the risk of bleeding. But even if you receive your treatment as prescribed, you may notice signs that your hemophilia treatment has stopped working as expected, which can happen if you have developed an inhibitor. Signs of hemophilia treatment failure can include:
- active bleeding that is increasingly difficult to control, even after receiving prescribed clotting factor treatments
- breakthrough bleeding symptoms in the joints or muscles that occur despite regular preventive clotting factor therapy, particularly if these bleeds are more frequent or severe than usual
Immediate next steps to take
If you experience symptoms that suggest hemophilia treatment failure, you may need to investigate the possible causes and consider other options. But not on your own. Working with a healthcare team that specializes in treating bleeding disorders is essential.
Consider contacting a Hemophilia Treatment Center (HTC) for a hematologist consultation. HTCs provide comprehensive care through a team of specialists, including physicians, nurses, physical therapists, and social workers, as well as education and support. Find an HTC in your area.
Ask for testing
The next step is to determine whether a hemophilia inhibitor is the reason your treatment has stopped working. A blood test called the Bethesda assay is one of the most common diagnostic tests used to detect and measure inhibitor levels. The results are measured in Bethesda units, which help clinicians determine whether a person has a low or high inhibitor titer.
People with a lower inhibitor titer have lower levels of inhibitors in the blood and may be managed by adjusting the dose or frequency of clotting factor treatment. Those with a high inhibitor titer will likely need to explore other treatment options. Your care team will recommend the best approach based on your test results, individual needs, and overall health.
After reviewing your test results and your treatment log, you can discuss what’s next. And you are as vital to the discussion as anyone on the team. Questions you can ask to start the discussion:
- The Bethesda assay test confirmed the presence of an inhibitor. What are the treatment options?
- Can we look at the convenience, risks, and long-term protection of these options?
- What are the pros and cons of making treatment changes or introducing new therapies to my plan?
Exploring your next treatment options
Changing your hemophilia treatment regimen requires research and partnership with your care team. If you have an inhibitor, your care team can help you explore treatment options beyond factor replacement therapy which can include:
- Bypassing agents: These therapies, also known as BPAs, trigger blood clotting through mechanisms that do not depend on the clotting factor being targeted by inhibitors. BPAs may be prescribed to manage active bleeding or prevent bleeds.
- Nonfactor therapies: These treatments also promote blood clotting without replacing the missing or dysfunctional clotting factors. There are two kinds of nonfactor therapies:
- Factor-mimicking treatments, which work similarly to the missing clotting factor
- Rebalancing agents, which reduce the activity of anti-clotting proteins to promote blood clotting
- Immune tolerance induction (ITI): The goal of ITI is to re-educate your immune system to prevent it from attacking clotting factors when they enter your blood. ITI generally involves administering factor replacement therapy frequently over a set period of time. If the body adjusts, doctors may have patients resume their usual factor replacement therapy.
As you weigh your options for a new treatment plan, keep in mind that it should align with your lifestyle and health goals.
Consider a second opinion
There are many reasons to get a second opinion. Maybe you aren’t comfortable with the plan your healthcare team proposed, you’re still experiencing bleeding episodes, or something just feels “off.” Whatever the reason, getting a second opinion on your care plan can help you decide what’s right for you.
If you suspect that your hemophilia treatment has stopped working, even though you’ve adhered closely to your treatment plan, you still have options. Partner with your healthcare team to address symptoms, evaluate the efficacy of your treatment, and update or change your treatment plan. The best hemophilia treatment plan is the one that helps you manage your hemophilia successfully.
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.
