Haemophilia A
Haemophilia is a rare, lifelong, inherited bleeding disorder characterised by spontaneous or traumatic bleeding caused by deficient levels of a coagulation protein: in haemophilia A this is factor VIII (FVIII).1
Beyond a Bleeding Disorder
There are approximately 8,800 people in the UK with Haemophilia A2: a rare and lifelong inherited bleeding disorder in which the blood does not clot properly, due to a lack of a protein called Factor Eight (FVIII). This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.
Before FVIII replacement therapy was available to treat and prevent bleeds, patients, predominately male, had a much-reduced life-expectancy versus the general population. However, since the introduction of intravenous FVIII infusion therapy in 1980s, particularly with the focus on preventative (prophylactic) treatment, the general life-expectancy is now near normal3.
Despite this improvement in the management of haemophilia, challenges continue to exist, with a drive towards the goal of achieving a “functional cure” via the following milestones: (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds; (d) Attainment of ‘normal’ mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis4.
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For full details and a summary of the adverse events please refer to:
→ HEMLIBRA Prescribing Information and Patient Information Leaflet – Great Britain
→ HEMLIBRA Prescribing Information and Patient Information Leaflet – Northern Ireland
→ Adverse event reporting and additional monitoring
Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to Roche Products Ltd. Please contact Roche Drug Safety Centre by emailing [email protected] or calling +44 (0)1707 367554.
As HEMLIBRA is a biological medicine, healthcare professionals should report adverse reactions by brand name and batch number.
References
- Oldenbury J, et al. N Engl J Med 2017; 377: 809-18
- UKHCDO annual report 2021 and bleeding disorder statistics for the financial year 2020/21, http://www.ukhcdo.org/home-2/annual-reports Accessed January 2023
- Franchini M, Mannucci PM. Orphanet J Rare Dis. 2012 May 2;7:24
- Skinner S, et al. Haemophilia. 2020; 26: 17-24
Abbreviations
FVIII, Factor VIII.
M-GB-00012641 | June 2023
