Hemophilia A Clinical Trial
Official title:
Evaluation of Real World Outcomes With Extended Half-Life Concentrates for Routine Clinical Use in Haemophilia A and B: UK - EHL Outcomes Registry
Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male
patients and are characterised by low levels of circulating clotting factors VIII and IX
respectively. Clinically low levels present with multiple recurrent bleeds into joints and
muscle from the first couple of years of life. In addition patients may present with
spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is
replacement with the missing factor in the form of intravenous injections of factor VIII and
IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and
the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and
aims to decrease spontaneous bleeding events and resulting joint damage, and this requires
patients to self-infuse factor into their veins two to four times week. Patient's compliance
with prescribed regimen and recommendations has a significant influence on outcomes.
Advances in biomolecular and protein engineering have extended the duration of the effect of
clotting factor VIII and IX through multiple mechanisms. This extension of the duration of
the effect presents the clinician and patients with opportunities to tailor the treatment to
their particular needs, circumstances and body other characteristics. It has been suggested
that decreasing the frequency of infusions will improve adherence and thus contribute to
improved outcomes.
In rare disorders, it is an accepted fact that post-marketing studies are crucial to
understand the generalisability of the efficacy and safety outcomes and identify any new
safety and efficacy concerns in relation to specific population group. The investigators
propose the development of a registry for systematic collection of information with the dual
aim of analysing the relationship between patient and treatment characteristics, and
outcomes, and simultaneously identify areas for practice development that can improve the
overall quality of life experienced by the haemophilia patient community.
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