Prophylactic therapy’s efficacy in improving the health of patients with haemophilia is now well known, to the extent that has come to be recognised as the strategy of election in the treatment of this condition. However, does the efficacy of prophylaxis translate into a tangible improvement in patients’ quality of life?
A recent study endeavoured to answer this question, by addressing, for the first time, the populations of haemophiliac patients who have the greatest difficulty coming to terms with the burden of the disease: children and teenagers. The Authors administered a questionnaire designed specifically to assess HRQoL (Health-Related Quality of Life), i.e. the impact that health has on a person’s wellbeing, according to their individual expectations.
The questionnaire was administered to 401 children and teenagers between 7 and 18 years of age from 9 European, American and Asian countries. The respondents also had haemophilia of varying severity (mild, moderate and severe), and were treated with both on-demand and prophylactic treatment.
The results of the study first and foremost confirmed that the prophylactic regimen was more efficacious than on-demand therapy, also in terms of improvements in quality of life. Furthermore, although it is true that the condition’s negative impact on quality of life increases with its severity, it is also true that the beneficial effects of prophylaxis follow the same trend. This means that, the more severe the patient’s haemophilia, the greater the benefits of therapy. The data even show that children and teenagers with severe haemophilia on prophylactic therapy express a similar degree of satisfaction with their life as patients of the same age with moderate haemophilia treated with on-demand therapy.
This study confirms that the evolution of haemophilia treatment is heading in the right direction, towards the goal of guaranteeing each patient not only a healthy life, but also one that is rewarding and happy at all ages.
Usuba K. et al, Impact of prophylaxis on health‐related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries. Res Pract Thromb Haemost. 2019; 3:397–404.