Hemophilia, a lifelong bleeding disorder caused by a deficiency in specific clotting factors, requires more than just awareness, it demands effective management to prevent complications and maintain quality of life.
While the condition can present serious challenges, advancements in treatment have significantly improved outcomes for those affected.
In this article, we explore the various treatment approaches for hemophilia, including both pharmacological therapies like clotting factor replacement and emerging gene therapies and, lifestyle modifications and preventive care.
Understanding these options is key to empowering individuals, caregivers and healthcare providers to manage hemophilia with confidence and care.
Types of Care: Episodic vs. Prophylactic
Healthcare providers typically prescribe treatment in two forms—episodic care and prophylactic care. Episodic care is used to stop bleeding when it occurs, while prophylactic care involves regular infusions to prevent bleeding episodes before they happen. With proper training, individuals with hemophilia can learn to self-administer infusions at home. This allows for quicker treatment, fewer complications, and greater independence.
1. Clotting Factor Products
There are two main types of clotting factor concentrates:
a. Plasma-Derived Factor Concentrates: These are made from donated human plasma. They undergo extensive screening and viral inactivation processes to ensure safety.
b.Recombinant Factor Concentrates: These are not made from human blood. Approved since 1992, they are created using DNA technology and offer a safer alternative with zero risk of transmitting bloodborne viruses.
However, some individuals may develop inhibitors, which are antibodies that neutralize the effect of the infused clotting factor. In such cases, higher doses or different treatment products may be needed.
2. Alternative and Supportive Treatments
◦ DDAVP® and Stimate® (desmopressin acetate): These medications stimulate the release of stored factor VIII in mild or moderate hemophilia A. DDAVP is given via injection, and Stimate is administered as a nasal spray.
◦ Amicar® (epsilon amino caproic acid): This antifibrinolytic medication helps stabilize blood clots, especially useful for mouth bleeds or dental procedures. It can be taken orally or via injection.
◦ Pain Management and Joint Care: Steroids, pain medications and physical therapy may be used to manage pain and swelling in affected joints.
3. Gene Therapy for Hemophilia
Recent advancements include gene therapy, which offers a potential long-term solution by delivering a working gene to help the body produce the missing clotting factor.
◦ Roctavian® is a gene therapy approved in 2023 for adults with severe Hemophilia A. It uses a modified virus to deliver the gene responsible for producing factor VIII.
◦ Hemgenix® is a gene therapy for Hemophilia B, approved in 2022. It helps the liver produce factor IX and is suitable for adults who require frequent prophylaxis or have a history of severe bleeding.
Gene therapy is administered via a one-time intravenous infusion. However, patients must be tested for immunity to the delivery virus (AAV5) before receiving this therapy.
4. Extended Half-Life and Non-Factor Therapies
a. Clotting factor products can also be classified by how long they last in the body:
◦ Standard Half-Life Therapies require more frequent dosing.
◦ Extended Half-Life Therapies have been modified to last longer, reducing the frequency of infusions.
b.Non-Factor Therapies like Hemlibra represent a newer class of treatment that doesn’t involve infusing clotting factor directly but instead works by enhancing the clotting process in other ways.
5. Home Treatment Benefits
Home treatment offers several benefits: quicker access to care, fewer hospital visits, cost savings and more control for patients and families. Vein access devices may be surgically implanted to ease frequent infusions, though they come with risks such as infections.
Choosing the Right Treatment
Treatment varies based on the severity of hemophilia:
◦ Mild Hemophilia: May only require DDAVP before surgery or physical activity.
◦ Moderate Hemophilia: Occasional replacement therapy or DDAVP for certain activities.
◦ Severe Hemophilia: Regular preventive replacement therapy is usually necessary, often started at a young age and continued for life.
Quick recognition and treatment of bleeding are critical to prevent joint damage and other complications. Families and caregivers should also be educated on how to spot the signs of internal bleeding, especially in children who may avoid reporting symptoms to escape treatment discomfort.
Looking Ahead
Researchers are continually working on improving therapies, reducing side effects like inhibitor development and expanding gene therapy access. Collaborating closely with a hematologist or a hemophilia treatment center ensures the best possible care tailored to individual needs.
Lifestyle Tips for Hemophilia Patients
Living with hemophilia requires attention to health, safety and emotional well-being. With the right lifestyle adjustments, individuals with hemophilia can lead full, active and independent lives. Here are essential tips to help manage your condition more effectively:4,5,6
1. Stay Active Safely
Regular exercise helps build muscle strength and joint stability, which reduces the risk of bleeds.
◦ Choose low-impact activities like swimming, walking or cycling.
◦ Avoid high-risk sports (e.g., football, wrestling) unless cleared by your doctor.
◦ Work with a physiotherapist specialized in hemophilia to create a tailored fitness plan.
◦ Always follow prophylactic treatment recommendations before engaging in physical activity.
2. Maintain a Hemophilia-Friendly Diet
Healthy eating supports overall well-being and joint health.
◦ Include: Lean protein, leafy greens, fresh fruits, vegetables and whole grains.
◦ Limit: Excessive vitamin E, omega-3 fatty acids and processed foods that may affect clotting.
◦ Work with a nutritionist to develop a personalized plan.
3. Stay Hydrated
Dehydration can affect blood flow and overall health.
◦ Aim to drink 6–8 glasses of water daily.
◦ Carry a water bottle to stay consistently hydrated.
4. Be Medication Smart
Medication adherence is key.
◦ Stick to your prescribed treatment schedule.
◦ Use reminder tools (e.g., apps, alarms, calendars).
◦ Keep a record of infusions and bleeds.
◦ Communicate openly with your care team about side effects or missed doses.
5. Prioritize Dental Health
Dental procedures can trigger bleeds.
◦ Maintain good oral hygiene to prevent issues.
◦ Inform your dentist about hemophilia in advance.
◦ Use prophylactic treatment or antifibrinolytics (e.g., Amicar) when necessary.
6. Know How to Handle Bleeds
Early recognition and prompt treatment are crucial.
◦ Signs of a bleed: swelling, warmth, tingling, stiffness, unexplained pain.
◦ Use RICE method: Rest, Ice, Compression, Elevation.
◦ Keep emergency clotting factor accessible at all times.
◦ Educate family and caregivers on first aid protocols for bleeding disorders.
7. Protect Your Mental Health
Chronic conditions can take an emotional toll.
◦ Practice mindfulness, meditation, or journaling.
◦ Don’t hesitate to seek help from a therapist or psychologist.
◦ Join support groups to connect with others who understand your journey.
8. Educate and Involve Family and Friends
Helping others understand your condition ensures better support.
◦ Explain your condition and signs of bleeding.
◦ Teach them how to respond in emergencies.
◦ Share resources and involve them in care routines when necessary.
9. Prepare for Emergencies
Have a plan in place in case of unexpected bleeds or disasters.
a. Create a Family Emergency Kit with:
◦ Clotting factor
◦ Emergency contacts
◦ Medical ID card or bracelet
◦ Treatment instructions
b. Ensure everyone in your household knows the plan.
10. Travel Smart
Travel is possible with the right preparation.
◦ Carry medications, supplies and medical documents in your carry-on.
◦ Research nearby hospitals or treatment centers at your destination.
◦ Avoid risky activities that could lead to bleeds while traveling.
11. Managing Hemophilia in Children Caring for children with hemophilia requires extra precautions.
◦ Educate schools, babysitters and caregivers about the condition.
◦ Develop a treatment and emergency response plan.
◦ Help children understand their condition and how to communicate symptoms.
12. Stay Informed About New Treatments Medical advancements are transforming hemophilia care.
◦ Learn about gene therapy, extended half-life products, and non-factor therapies.
◦ Discuss new treatment options regularly with your hematologist.
Conclusion
Managing hemophilia effectively requires a comprehensive approach that includes timely treatment, informed choices, and proactive lifestyle adaptations. Thanks to advances in medicine ranging from extended half-life therapies to revolutionary gene therapies, individuals with hemophilia today have more options and greater hope than ever before. However, successful management goes beyond medical care; it involves education, awareness and support from families, caregivers and healthcare professionals. By staying informed, being medication smart and prioritizing both physical and emotional well-being, people living with hemophilia can lead active, fulfilling lives. As research continues to evolve, the future holds even greater promise for improved care and lasting solutions. Empowerment through knowledge is the key because with the right care and support, hemophilia doesn’t have to define your life.
References
- Hematology-Oncology Associates of CNY. How Is Hemophilia Treated? Treatment With Replacement Therapy.HOA. Accessed on April16, 2025. https://www.hoacny.com/patient-resources/blood-disorders/what-hemophilia/how-hemophilia-treated
- National Bleeding Disorders Foundation. Current treatments. National Bleeding Disorders Foundation. Accessed on April16, 2025. https://www.bleeding.org/bleeding-disorders-a-z/treatment/current-treatments
- Treatment of Hemophilia. CDC. Published on November 13, 2024. Accessed on April16, 2025. https://www.cdc.gov/hemophilia/treatment/index.html
- Hemophilia Outreach Center. Coping with Bleeding Disorders: Practical Lifestyle Tips for Daily Management. Hemophilia Outreach Center. Published on November 1, 2024. Accessed on April 6, 2025. https://hemophiliaoutreach.org/coping-with-bleeding-disorders-practical-lifestyle-tips-for-daily-management/
- Living with hemophilia. CDC. Published on June 27, 2024. Accessed on April16, 2025. https://www.cdc.gov/hemophilia/living-with/index.html
- The Haemophilia Society. Healthy Living. The Haemophilia Society. Accessed on April16, 2025. https://haemophilia.org.uk/support/day-day-living/healthy-living/
