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Coagulation Disorders Haemophilia
Figure 1: Boys with Severe Haemophilia Experience Significantly
western Europe showed that not only did prophylaxis improve
Fewer Bleeding Episodes with Prophylaxis Compared with
orthopaedic outcome, but a survey of these patients showed a high
Episodic Treatment
level of health-related quality of life as well.
14
Prophylaxis in children with haemophilia therefore presents a
1.8 n=25
1.6
beneficial outcome in terms of preventing joint damage, reducing the
1.4
n=31
n=31 risk of inhibitor development and, as a result, improving overall quality
n=33
1.2
n=24 of life, allowing children to be children and to live the lives they
1.0
choose. The use of recombinant concentrates not only maintains these
0.8
.
haemorrhages/month
0.6
n=23
advantages, but offers the safest available option in factor
0.4 n=31 n=26
n=29 n=29
replacement therapy.
Average no
0.2
0.0
12345 Prophylaxis in Adults – Benefits and Challenges
Age (yr)
Today’s population of adult patients with severe haemophilia can be
Prophylaxis: Prophylaxis: Episodic therapy: Episodic therapy:
categorised into two groups: those in whom treatment has been
joint haemorrhages other haemorrhages joint haemorrhages other haemorrhages
sufficient and those in whom treatment has been insufficient, i.e.
Source: Manco-Johnson et al., 2007.
12
those who have received early prophylaxis and those who have not. Dr
Jørgen Ingerslev of Denmark spoke of his own experiences with adults
In this respect, the potential for unknown blood-borne infections who had received no early prophylaxis; many are now disabled to
remains a valid possibility. In comparison, the recombinant various degrees due to repeated joint and muscle bleeds and today
concentrates resulting from genetic engineering are the safest suffer from chronic joint disease, and have had to undergo
available alternative in factor replacement therapy, and their usage orthopaedic joint surgery. However, the majority of patients who had
should be encouraged. received early prophylaxis – of dosages varying between the Swedish
and Dutch dosage schemes – now have almost completely normal joint
Allergic reactions to prophylactic FVIII have the potential to be quite function. A few still experience minor difficulties with the weight-
severe, where the patient may have to withdraw from using the bearing ankle joints. Therefore, it appears that prophylaxis plays a
particular concentrate. Fortunately, safety and efficacy studies have significant role in preventing disability.
shown that rFVIII can have a rate of allergic reaction as low as 0.004
per infusion.
10
There are four different types of prophylaxis for adults. Some may
choose to continue on their prophylactic treatment from childhood.
In spite of the aforementioned issues with prophylaxis, the primary Others may also choose to continue secondary prophylaxis, utilising
concern for physicians nowadays is undeniably the development of the same principles as adopted in childhood and adolescence.
inhibitors. Much research has focused on this aspect; studies have Alternatively, periodic prophylaxis may be chosen for a certain period
shown that although there are elements that can increase the risk of of time in order to prevent recurrent bleeds or joint pains caused by
inhibitor development,
9,11
there is a notable benefit for prophylaxis, synovitis. Lastly, patients can choose an on-demand, self-adjusted
where the risk of inhibitor development is 60% lower compared with treatment regimen of episodic prophylaxis.
that seen with on-demand treatment.
11
Prophylaxis in adults with severe or moderate haemophilia is growing in
Benefits of Prophylaxis popularity. A survey of 21 haemophilia centres in Europe showed that
The obvious benefit in adopting a prophylactic regimen is the approximately 35% of adolescent patients currently continue on their
reduction of joint bleeds. Recently, the Joint Outcome Study has treatment from childhood prophylaxis, with 39% able to successfully
shown that prophylaxis, compared with enhanced on-demand reduce or discontinue their treatment programme for a short period of
treatment, was associated with an annual reduction of 87% in joint time; 30% of patients discontinue treatment permanently.
15
Nearly
bleeds (p<0.001) and an annual reduction in the total number of 20% of patients over 50 years of age currently receive some form of
bleeds by 81.5% (p<0.001) (see Figure 1).
12
Furthermore, structural prophylaxis, and this number is gradually increasing.
joint damage as determined by magnetic resonance imaging (MRI)
occurred in only 7% of boys on prophylaxis compared with 45% of Benefits and Evidence of Prophylaxis
boys receiving on-demand therapy (p<0.002). The goal of prophylaxis is to improve a patient’s health and general
wellbeing, with the ultimate objective of improving overall quality of life. In
The benefits of prophylaxis are evident once the regimen is initiated. the younger patient, the aim of prophylaxis is to prevent joint- and muscle-
Using the Czech Republic as an example, Dr Blatny explained that all˘ related disabilities. This can reduce the number of days away from school or
children with severe haemophilia began prophylactic treatment with work, or allow the patient to participate in sports and exercises, thereby
factor concentrates under the Dutch model in 1991. Seventeen years improving quality of life. In the older patient who has not received previous
later, these Czech children with haemophilia – even the 2.6% with prophylaxis, prophylactic treatment may offer an opportunity to reduce the
inhibitors – experience a median of only 4.5 bleeds per year.
13
This likelihood of further disability. Although unlikely to fully reduce the amount
should be a prime example for countries that still do not use of pain in the patient, it is likely that prophylaxis can instigate some
prophylaxis routinely, as these dramatic differences can emerge within reduction, allowing patients to take better care of themselves, therefore
15–18 years. Indeed, a study of children four to 16 years of age in improving their quality of life.
22 EUROPEAN HAEMATOLOGY
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