Sumit Parikh is the AHCDO ABDR Senior Research Fellow
Prophylaxis is considered standard of care in all children with severe haemophilia. However, the timing of commencement, dosing regimen and continuation of prophylaxis into adulthood varies for each individual patient.
Last year AHCDO conducted a large retrospective study characterising current practice with regards to the use of prophylactic clotting factor infusions in patients with haemophilia A and haemophilia B in Australia.
Overall the proportion of patients receiving prophylaxis (according to disease severity and age) compared well with other developed nations. Prophylaxis in children and young adolescents with severe haemophilia A and haemophilia B was near universal, which is an impressive achievement and cast a positive reflection on the delivery of multidisciplinary haemophilia care in Australia.
Adherence to prophylaxis among different age groups was also evaluated as part of the study and was determined by the ratio of observed to expected factor usage. Young adults were the most non-adherent group to prophylaxis and this was clearly evident from the results. As a consequence of this, despite patients being able to self-administer factor, non-adherence prevents them from achieving the real benefit of prophylaxis treatment (reducing bleeds and better quality of life).
This study provided a valuable insight into the current prophylaxis practice in Australia plus an opportunity to take this further and formally assess the real benefit of prophylaxis.
The biggest challenge to formally assess the real benefit of prophylaxis is the need to actively monitor bleed outcome. MyABDR is a secure app for smartphones and a website for patients with bleeding disorders to self-record their home treatments and bleeds. However, the uptake of MyABDR has been disappointingly low, which means that a reliable assessment of how effective the prophylaxis treatment has been for patients cannot be determined. There are a lot of resources and support material available for patients and their caregivers to assist in using the MyABDR application and its features and it is in everyone’s best interests to take advantage of this and make the most of the application. This is because the usefulness of the ‘MyABDR’ application is not restricted to self-assessment but it also builds the knowledge base to be able to determine the effectiveness of various treatments provided to patients with bleeding disorders. The community could gain enormously with individualised prophylaxis, emerging extended half-life products and other novel treatment regimes, however this cannot be ascertained without being able to formally assess the treatment outcomes.
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