Reflection of Progress in Hemophilia Treatment in Current WFH Recommendations
In June 2020, the new, third guidelines of the World Federation of Hemophilia (WFH) were introduced. They are based on the fact that in the past 5 years there has been significant progress in the management of hemophilia, mainly due to the introduction of new − even revolutionary − drugs into clinical practice.
Introduction
Besides new factor preparations with extended half-lives, the first representative of non-factor therapy (i.e., ensuring a certain level of hemostasis by a different method than the administration of the missing factor), emicizumab, has also entered clinical practice. It is a bispecific monoclonal antibody that mimics the function of factor VIII as a cofactor. With the use of emicizumab, hemostasis corresponding to approximately 15% FVIII levels is achieved, representing mild hemophilia A. Its advantages also include subcutaneous administration and a lower frequency of dosing.
Several Factual Notes on Emicizumab in Current WFH Recommendations
- The administration of new drugs requires patient education, their families, and supervision during the transition to the new type of treatment.
- Emicizumab can become home treatment after previous training in subcutaneous administration.
- Emicizumab has a long biological half-life and high effectiveness in preventing bleeding episodes.
- Emicizumab is not intended for the treatment of acute bleeding.
- The subcutaneous application of emicizumab simplifies the initiation of possible prophylaxis in children at an early age, without the need for a central venous catheter. However, there is currently limited data and additional information for children under 1 year of age.
Key Points from WFH Recommendations for Clinical Use of Emicizumab
5.7.1
For patients with hemophilia A and an inhibitor, emicizumab should be used for regular prophylaxis.
For patients with hemophilia A without an inhibitor, emicizumab may be used for regular prophylaxis.
6.5.1
For patients with severe hemophilia A without an inhibitor, prophylaxis with emicizumab will prevent hemarthrosis. However, there is little data regarding long-term follow-up, and it is therefore important to obtain these data.
8.3.7
Emicizumab is used for prevention, not for treating bleeding. In the case of bleeding in patients with an inhibitor, replacement therapy is needed.
For patients with an inhibitor and in the event of bleeding, the use of rFVIIa is preferred, as treatment with aPCC (in combination with emicizumab) is associated with the risk of developing thrombotic microangiopathy (TMA).
Caution is also needed for patients treated with emicizumab in combination with rFVIIa and simultaneously with risk factors for thrombosis (history of venous thromboembolism, obesity, smoking, chronic infection, inflammation), as these patients are at risk of acute myocardial infarction (non-STEMI) and pulmonary embolism (PE).
8.3.17
For patients with hemophilia A and persistent inhibitor, who have failed immune tolerance induction (ITI) or have never undergone it, emicizumab is preferred over bypassing agents, as it is more effective in preventing bleeding and easier to administer.
8.3.19
For patients with severe hemophilia A and an inhibitor, emicizumab is recommended over prophylaxis with bypassing agents to reduce bleeding episodes.
(eza)
Did you like this article? Would you like to comment on it? Write to us. We are interested in your opinion. We will not publish it, but we will gladly answer you.
