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Published on: September 30, 2021
Prevention and Management of Bleeding Episodes in Children with Hemophilia
1Department of Clinical Sciences-Paediatrics, Lund University, Lund, Sweden. Rolf.Ljung@med.lu.se.
Insights
Hemophilia treatments have advanced with extended half-life (EHL) factor concentrates and novel therapies like FVIII mimetics. Gene therapy also shows promise for hemophilia A and B.
Area of Science:
- Hematology
- Biotechnology
- Pharmacology
Background:
- Hemophilia A and B are historically managed with frequent intravenous factor replacement therapy.
- While recombinant factor concentrates have improved care for over 25 years, frequent administration remains a challenge.
- Extended half-life (EHL) formulations aim to reduce treatment burden.
Purpose of the Study:
- To review advancements in hemophilia treatment, focusing on extended half-life products and novel therapeutic strategies.
- To highlight the benefits and limitations of current and emerging hemophilia therapies.
- To discuss the potential of gene therapy as a future treatment modality.
Main Methods:
- Review of literature on recombinant factor VIII (FVIII) and factor IX (FIX) concentrates.
- Analysis of extended half-life (EHL) technologies including albumin fusion, Fc-fusion, and PEGylation.
- Examination of novel treatments such as FVIII mimetics and monoclonal antibodies.
- Overview of gene therapy approaches for hemophilia.
Main Results:
- EHL-FIX demonstrates significant half-life extension (3-5 times), while EHL-FVIII shows a more modest improvement (1.5 times).
- Novel therapies offer long-lasting activity, subcutaneous administration, and efficacy in antibody-positive patients.
- Gene therapy represents a significant advancement with the potential for a curative approach.
Conclusions:
- Extended half-life factor concentrates and innovative therapies are improving hemophilia management by reducing administration frequency.
- FVIII mimetics and monoclonal antibodies provide alternative treatment options with distinct advantages.
- Gene therapy holds great promise for the future treatment of hemophilia A and B.
Abstract:
Regular prophylactic treatment with factor VIII (FVIII) and factor IX (FIX) concentrates in hemophilia A and B, respectively, is introduced in early infancy and has resulted in dramatic improvement of the conditions. Recombinant FVIII and FIX concentrates have been available for > 25 years and have been modified and refined through the years; however, unfortunately frequent intravenous administrations are still necessary. The half-lives of these products have now been extended (EHL) by fusion with albumin, the Fc-portion of IgG, or by being PEGylated. This has been very successful for EHL-FIX, with 3-5 times longer half-life, and to a lesser degree for EHL-FVIII with a half-life extension of only 1.5 times the conventional products. New treatment principles using FVIII mimetics or monoclonal antibodies that rebalance the pro- and anti-coagulation system by interfering with production of anti-thrombin or tissue factor pathway inhibitor have the benefits of long-lasting activity, subcutaneous administration, and being useful in patients both with and without neutralizing antibodies. As the ultimate treatment, recent progress has also been made with gene therapy of both hemophilia A and B.
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