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Non-factor replacement therapies

Information correct as of July 2019

Regular prophylaxis of factor replacement has been the mainstay of treatment for people with haemophilia. Factor prophylaxis helps the blood to clot and can minimise the likelihood of bleeds. A typical prophylaxis treatment regimen can involve two or more intravenous (into a vein) infusions a week (1).

There is also research ongoing to look at different approaches to managing haemophilia. This includes the development of treatments that are not clotting factors and work differently to replacing FVIII or FIX (1).

Some of the treatments in development are made from proteins or small molecules. These are given subcutaneously (under the skin) and look to replace the activity of the missing clotting factor, or to re-balance clotting capacity (2).

Emicizumab (which is also called Hemlibra®▼) is the only non-factor replacement therapy currently available by prescription in the UK. It is approved for people of all ages who have congenital haemophilia A with FVIII inhibitors, or severe congenital haemophilia A (FVIII <1%) without FVIII inhibitors (3).

Emicizumab is a prophylactic treatment given subcutaneously (3). It works by restoring the function of missing activated FVIII, that is needed for effective blood clotting. It is used to prevent bleeding or reduce the number of bleeding episodes but must not be used to treat a bleed (4).

Emicizumab is administered once weekly for the first 4 weeks (this is called the loading phase). Thereafter, it can be given either once weekly, once every 2 weeks or once every 4 weeks (called the maintenance phase). The amount of emicizumab required is dependent on a person’s weight (4).

Clinical trials have been undertaken to evaluate the safety and efficacy of emicizumab (5-8). Like all medicines, emicizumab can cause side effects, although not everybody gets them. In clinical trials, serious side effects were seen in people using activated prothrombin complex concentrate (aPCC) whilst receiving emicizumab. Use of aPCC should be avoided in people receiving emicizumab, unless no other treatment options are available (4).

Reporting of side effects

▼This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get.

If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any possible side effects not listed in the package leaflet. You can also report side effects directly via the Yellow Card Scheme at www.mhra.gov.uk/yellowcard, or sear for MHRA Yellow Card in the Google Play or Apple App Store. You should also report side effects to Roche Products Ltd by emailing the Roche Drug Safety Centre at Welwyn.uk@dsc@roche.com or calling +44 (0) 1707 367554. By reporting side effects you can help provide more information on the safety of this medicine.  

References:

  1. Weyand, A. C., & Pipe, S. W. New Therapies for hemophilia. Blood, (2018), blood-2018-08-872291. Accessed July 16, 2019. https://doi.org/10.1182/blood-2018-08-872291
  2. Carr ME, Tortella BJ. Emerging and future therapies for haemophilia. J Blood Med, 2015 Sep 3;6:245-55.
  3. Hemlibra summary of product characteristics https://www.medicines.org.uk/emc/product/9043/smpc Accessed July 2019
  4. Hemlibra patient information leaflet https://www.medicines.org.uk/emc/product/9043/pil Accessed July 2019
  5. Oldenburg J, et al. N Engl J Med 2017; 377: 809-18
  6. Young G, et al. ASH 2018 [oral presentation 632]
  7. Mahlangu J, et al. N Engl J Med 2018; 379: 811-22
  8. Pipe S, et al. Lancet Haematol 2019. Published online http://dx.doi.org/10.1016/S2352-3026(19)30054-7