haematology and oncology
Sponsored
Evolving protection
in haemophilia
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
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
Support for the
preparation of this
report was provided
by Swedish Orphan
Biovitrum
20
15
10
5
0
Belgium
n=28
France
n=35
Germany
n=106
Italy
n=111
Spain
n=20
Sweden
n=94
UK
n=188 b
a
n numbers are shown for overall number of subjects (severe an d moderate) in each country; b annualised bleeding rates for the UK are based on patients’
self-reported bleedings in Haemtrack
13
HHE 2018 | hospitalhealthcare.com