In someone without haemophilia, these happen all the time and normally go unnoticed; a small blood vessel might break just from regular movement, but their body can easily control any bleeding. In someone with haemophilia, the bleeding may continue and this can lead to irreversible damage to joints.1
“Microbleeds have therefore become a key concern for the haemophilia community,” says Dr Gallia Levy, Former Associate Group Medical Director at Roche. “Even one bleed will cause some damage, so consistent and effective treatment is needed.”
“We’re in the midst of a very interesting time in haemophilia research,” she says. The change from on-demand treatment when a bleed occurs to prophylaxis to prevent bleeds was an important shift in haemophilia care. She hopes that achieving consistent protection over time is the next step.
Prophylaxis – now considered the standard of care in haemophilia A treatment – can on average, cut down bleeds from more than 40 times a year to less than two bleeds a year.2 Prophylaxis can protect against spontaneous bleeds, such as joint bleeds, as well as potentially life-threatening bleeds such as those in the head or gastrointestinal tract.3 Adherence to a prophylaxis protocol is critical to its success to maintain appropriate clotting levels in the blood.4
People with haemophilia A usually need to administer their treatment every few days – or as often as every day for those who have developed inhibitors – which may be burdensome to patients or their caregivers.5 “Worrying about the potential for a bleed can become emotionally burdensome,” Levy explains.
Factor VIII replacement therapy is short-acting and needs to be administered frequently to ensure bleed protection. This is because the half-life of the replaced factor VIII, or the amount of time it takes for factor levels to drop by 50%, is approximately 8-19 hours for factor VIII replacement therapy.6
People with haemophilia A often adjust their schedule so that they administer treatment on the days when they are going to have the most activity, to minimise concern over whether they are adequately protected.
Haemophilia A is a life-long condition, the impact of which is felt from a young age. Finding a vein three times a week for treatment infusions can be difficult, especially in children. This means preventative treatment often doesn’t begin before the age of one to one and a half, according to Dr. Levy. However, in the time that these infants are not getting preventative treatment, they are at risk of bleeds.8
“There is this period of risk of significant bleeds, like a head bleed or a gastrointestinal bleed, that people are just accepting right now because it can be very challenging to do prophylaxis in somebody that young,” says Dr Levy.
Additionally, these infants may experience bleeds that cause long-term damage to joints. “The case for starting prophylaxis as early as possible to avoid any joint damage is clear,” she says.
At the moment, prophylaxis offers protection from bleeds, but there are still peaks and troughs in that protection. According to Dr Levy: “Ultimately, people with haemophilia A should be able to feel that they are protected beyond the minimal amount, and that this protection is sustained so they can live each day free from anxiety over their risk of bleeding.”
A rare bleeding disorder in which the blood does not clot properly, due to a lack – or insufficient levels – of a clotting protein called factor VIII, haemophilia A can lead to uncontrolled and often spontaneous bleeding.9 Standard treatment is factor VIII replacement therapy, which can be given on-demand as needed, or as prophylaxis to prevent bleeds. Since the introduction of prophylaxis in the 1990s, people with haemophilia A have seen a dramatic reduction in the frequency of bleeds, which has had a huge impact on their day-to-day lives.10
References
Van Meegeren M. et al. Blood-Induced Joint Damage: The Devastating Effects of Acute Joint Bleeds versus Micro-Bleeds. Cartilage. 2013 Oct; 4(4): 313–320.
Valentino L et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012 Mar; 10(3): 359–367.
Makris M. Prophylaxis in haemophilia should be life-long. Blood Transfus. 2012 Apr; 10(2): 165–168.
World Hemophilia Foundation [Internet; cited May 2018]. Available from:
Elder-Lissai A, Hou Q, Krishnan S. The Changing Costs of Caring for Hemophilia Patients in the U.S.: Insurers’ and Patients’ Perspectives. Presented at: American Society of Hematology Annual Meeting; December 6-9, 2014; San Francisco, CA. Abstract # 199.
Cafuir L, Kempton C. Current and emerging factor VIII replacement products for hemophilia A. Therapeutic Advances in Hematology. 2017; 8(10): 303-313.
Ljung R. The risk associated with indwelling catheters in children with haemophilia. British Journal of Haematology. 2007; 138: 580–586.
Lee C, Berntorp E and Hoots K. Textbook of Hemophilia. 3rd ed. Wiley; 2014.
WFH. Guidelines for the management of hemophilia. 2012. [Internet; cited 2017 November]. Available from:
Nation Hemophilia Foundation [Internet; cited May 2018]. Available from:
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