For families across India living with haemophilia, a genetic disorder that causes uncontrolled bleeding, the journey has often been one of extreme caution. However, today the conversation is shifting from “how do we stop a bleed?” to “how do we prevent one from happening?” This is where prophylaxis management of haemophilia is gaining importance.
Normally, when an injury occurs, proteins in the blood called clotting factors work together to form a seal (a clot) and stop the bleeding. In people with haemophilia, one of these clotting factors, usually Factor VIII or Factor IX, is either missing or not working correctly. Thus, bleeding lasts much longer.
While external cuts are visible, the real concern is internal bleeding into joints like knees, elbows, and ankles. Over time, these repeated bleeds can lead to permanent joint damage and chronic pain. If untreated, they can affect the patient’s movements, such as the ability to walk and climb stairs. It can also negatively impact social and daily activities and disrupt both work and education.
Shift to prophylaxis
Traditionally, the standard of care in haemophilia has been ‘on demand’ treatment, injecting the factor concentrate to stop a bleed after it occurs. However, modern haemophilia management has moved towards prophylaxis. This involves regular infusions of clotting factor to keep levels high enough in the blood to prevent bleeds before they start. By maintaining a steady level of factors, patients can:
• Prevent spontaneous joint bleeds.
• Significantly reduce the risk of complications such as long-term joint damage.
• Engage in physical activities and daily life with much less anxiety.
Innovation of extended half-life (EHL) factors
One of the biggest hurdles for patients on prophylaxis has been the frequency of injections. Standard factor treatments often require infusions every second or third day because the half-life (the time the medicine remains active in the body) is relatively short. This is where EHL factors are changing the landscape. EHLs are made by modifying the factor protein to stay in the bloodstream for longer. These treatments offer two major benefits:
1. Fewer infusions: Patients may require fewer infusions per week, which is a significant relief, especially for children and their caregivers.
2. Consistent protection: Because the medicine lingers longer, there are fewer gaps in protection, giving patients a more reliable safety net, and reducing complications.
New, easy-to-use pen devices are making home treatment of haemophilia much simpler by replacing hospital-based infusions with pre-filled, portable options. This innovation reduces the stress of hospital visits, helping patients stay on track with their prevention plan while enjoying more freedom in their daily lives. For the Indian haemophilia community, the introduction of EHL treatments through doctor-led care translates into convenience as well as freedom. It means a child can attend school regularly without need for regular hospital visits or play a game of cricket with friends without worrying about an internal bleed.
With modern prophylaxis and the evolution of long-acting factors, haemophilia becomes a manageable part of a patient’s life, offering relief to both the patients and their families. To know more about these prophylactic treatment options, speak to your haematologist.
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