EHL preparations have the potential to change the quality of life for people with hemophilia

Introduction

Hemophilia A and B are congenital, X-linked bleeding disorders, in their most severe forms associated with recurrent bleeding, often without a known cause. The preferred treatment modality for these patients is so-called replacement therapy, which involves the administration of concentrates of the missing factor VIII (FVIII) in the case of hemophilia A and factor IX (FIX) in hemophilia B. In severe forms of the disease, or bleeding phenotypes of milder hemophilia, preventive treatment - prophylaxis - is often indicated, providing protection against bleeding. Newer drugs, so-called extended half-life (EHL) preparations, were developed to further improve this protection.

Benefits of EHL concentrates in a nutshell

For hemophilia A, the biological half-life of FVIII-EHL is approximately 1.5-1.6 times longer than that of original standard half-life (SHL) preparations. For FIX-EHL, it is even up to approximately 5 times longer compared to SHL. These new concentrates have the potential to extend dosing intervals in prophylactic administration, improve overall protection by achieving higher so-called trough levels, and facilitate patient adherence, especially for those with poorer peripheral venous access.

Positive impact on the life of a hemophiliac

Achieving higher trough levels can be beneficial for physically active hemophiliacs. It has already been defined that physical activity is necessary for hemophiliacs and is generally highly encouraged, especially when administering prophylaxis. Physical activity and an active lifestyle can have a very positive impact on the acceptance of not only the disease itself but also the necessary treatment. For many hemophiliacs, the possibility of improved protection allowing for safer engagement in sports activities is a reason to switch to EHL therapy. Knowing the pharmacokinetic profile is advantageous for confirming the therapy effect and further personalizing prophylactic dose adjustments, which allows for additional treatment regimen modifications.

Conclusion

The growing experience with EHL preparations in real clinical practice confirms that these newer drugs have great potential to influence the quality of life for people with hemophilia, their social engagement, and to facilitate their acceptance of the treatment itself.

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Source: Lambert T., Benson G., Dolan G. et al. Practical aspects of extended half-life products for the treatment of hemophilia. Ther Adv Hematol 2018; 9 (9): 295-308, doi: 10.1177/2040620718796429.