A Survey on Prophylaxis versus On Demand Therapy in Young Adults with Haemophilia in four European Countries.

Prophylaxis is widely accepted as the optimum standard of care for children with severe haemophilia. However, there is still a lot of discussion and debate on the necessity of prophylaxis continuing into adulthood. Prophylaxis has been the standard of care for children and adults inSweden for over 25 years. Therefore young adults in Sweden with severe haemophilia would, in the vast majority of cases, have been on prophylaxis since a very early age and this would have continued into adulthood. In contrast young adults with haemophilia in countries such asIreland, UK and France would generally be treated with on demand therapy, although some would be on prophylaxis. However, in these countries prophylaxis would have been introduced some time in the last 10 to 15 years, and therefore these young adults would not have grown up with the benefits of prophylaxis and would have started their lives with on demand therapy.

Declan Noone and I undertook a survey of young adults with severe haemophilia in four European countries: Sweden, France, UK and Irelandwith the co-operation of the respective countries. A total of 58 young men with haemophilia between the ages of 20 and 35 were interviewed by phone. They were asked questions in relation to how long they had been on prophylaxis, about target joints, major bleeds and mobility problems. They were also asked to answer a very simple questionnaire with five questions (EQ5D). The EQ5D questionnaire is based on questions related to mobility, self care, ability to carry out usual activities, levels of pain and discomfort, and levels of anxiety and depression The questionnaire allowed us to extrapolate a utility value for the quality of life on a scale from zero to one. Zero would correspond to death and one would correspond to a perfect quality of life.

The results of the survey are crystal clear. The benefits to the adults inSweden of having been treated prophylactically from an early age and continuing into adulthood were obvious. People with severe haemophilia in Sweden reported less target joints, less major bleeds and significantly lower mobility problems when compared to people with haemophilia in the other three countries. The average number of bleeds per year in Swedenwas just over three whereas, in the other three countries it varied from 16 to 20 bleeds per year. Only 25% of those in Sweden had target joints, whereas 94% to 100% of those in the other three countries had target (damaged) joints. Of the 58 individuals surveyed only 16 had no target joints. Of these 16, all except one are living in Sweden. There was a very clear differential in relation to the number of days missed from work or college. In Sweden the average number of days missed per person per year was 0.5, in Ireland it was five, in the UK it was 6.6 and in France it was 15. The figure in France is very high because the French persons with haemophilia questioned for the survey included two young men with haemophilia who had recently undergone joint replacement surgery and therefore they were missing a significant amount of college or work. This is not unusual as the requirement for orthopaedic surgery and joint replacement surgery will be there for individuals who don’t have access to prophylactic therapy. There is also a dramatic difference between Swedenand the other three countries when it came to the respondents views of their quality of life as measured by the EQ5D questionnaire. The average quality of life in the UK, Ireland and France varied from 0.68 to 0.74, whereas the average quality of life in Sweden was 0.93. This Swedish score is very close to a perfect quality of life (1.0) whereas those in the other three countries have the perception that their quality of life is about 25% lower than those in Sweden.

When we looked at the data in relation to the treatment regime it was very clear that the differences were not due to nationality or other factors. They were due to the fact that the individuals in Sweden had been on prophylaxis from a very early age and this was continuing into adulthood. This study is a small scale study with a relatively small number of participants. It would be interesting to gather more data from more countries and in particular it would be interesting to look at data from countries which use different prophylactic regimes. However, the benefits of long-term prophylaxis continuing into adulthood are clear from this survey. The improvement in quality of life, in ability to take part in society, in ability to attend college or work and not be limited by bleeding episodes or joint damage in Sweden is startling. Out of the 58 patients surveyed only 16 had no target joints, 15 of the 16 are living in Sweden. We hope to extend this survey to other countries and to continue this work in the future.

At this particular time, when budgets are coming under threat, when treatment regimes are been questioned, when high cost but high value treatment regimes such as prophylaxis are under threat in some countries it is timely to have this reminder of the transformation of quality of life which can be brought about by giving a person with severe haemophilia optimum therapy on an ongoing basis. Parents of children with haemophilia in Ireland whose children are treated using prophylaxis are also perhaps getting a glimpse into the future with this survey. If your child complies with his therapy and takes his prophylaxis regularly, as a young adult there is every reason to believe that his quality of life should be near perfect and he should be able to fully participate in college, in employment and in the everyday normal activities of life with no impediment.

We greatly appreciate the participation of the 58 people with haemophilia who responded to the survey. Your participation in the collection of this type of data is vital in assisting us as a haemophilia society to make the cogent arguments required to defend, maintain or improve the high standard of haemophilia care which we will always advocate for on a national basis.

Brian O’Mahony

Chief Executive