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A report from a satellite symposium held at the 11th Annual Congress of the European Association for Haemophilia and Allied Disorders in February 2018 , and sponsored by Swedish Orphan Biovitrum

Support for the preparation of this report was provided by Swedish Orphan Biovitrum

Long gone are the days when patients with haemophilia had to inevitably suffer the devastating consequences of this disease , which included life-threatening bleeding , pain , and disability . With the increasing adoption of coagulation factor replacement therapies , prophylaxis outcomes have substantially improved for patients , not only clinically but also in terms of quality of life ( QoL ). However , limitations to the long-term effectiveness and ability to prevent complications still persist with conventional anti-haemophilic coagulation factors . New coagulation factor replacement therapies with extended half lives that aim to provide individualised approaches and that are , at the same time , reducing treatment burden for patients , as well as being cost-effective , are now available . Indeed , the cornerstone of treatment remains preventing and controlling bleeding , and the current treatment paradigm is evolving , broadening its scope to include a more ‘ holistic ’ perspective of the disease , where joint care , pain control and QoL play a significant role .

In the past , the most common treatment regimens for haemophilia A involve the administration of factor VIII coagulation factor 3 – 4-times a week , 1 but a 12-month retrospective study in Europe showed that many patients still presented moderate-to-severe bleeding episodes , with median annualised bleeding rates ( ARBs ) ranging from 1.0 to 8.0 for patients on prophylaxis , and from 4.5 to 18.0 for those treated on demand . 2 Similar findings can be observed in patients with haemophilia A and B ( Figure 1 ). 2 The data demonstrate that there is still an unmet need with regards to offering protection against bleeding .

Extended half-life factors can help support prolonged haemostatic protection . Recombinant human coagulation factor VIII Fc fusion protein ( rFVIIIFc ) is an extended half-life FVIII molecule ( Elocta ® ; approved in 2015 for the treatment of figure 1

Annualised bleeding rates in patients with haemophilia A and B in several European countries a

Belgium Median ABR – all types of bleeds ( IOR )

20

15

10

5

a n numbers are shown for overall number of subjects ( severe and moderate ) in each country ; b annualised bleeding rates for the UK are based on patients ’ self-reported bleedings in Haemtrack

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