WFH NETWORK

Non-factor Replacement Hemophilia Therapies

 
Details

Year: 2019

Language: English

Author(s): Adapted from Global NMO Training 2018 plenary presentation by Glenn Pierce

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Additional Information

Adapted from: Global NMO Training 2018 plenary presentation by Glenn Pierce
Reviewed by: Glenn Pierce
Edited by: Georghia Michael

The learner is strongly advised to complete Module 1 Introduction to Bleeding Disorders and Module 2 Evolution of Treatment Products, in the WFH Treatment Products eLearning Program before embarking upon Module 4.

This fourth module in the Program introduces the treatment of bleeding disorders through strategies other than replacing the factor that is missing or doesn’t work correctly. Building upon the understanding of how clotting factors work in the coagulation cascade, and how a deficiency in a specific clotting factor can disturb the hemostatic balance, such as in hemophilia A and B, established in modules 1 and 2, this module explains how other points in the coagulation cascade are being targeted to find ways to re-balance hemostasis.

Substitution therapy is explained with the example of the single such product that has received regulatory approval, at the time of writing of this module, emicizumab. The principles of intervening at other points in the cascade are explored through the examples of tissue factor pathway inhibitor (TFPI) and antithrombin (AT). The bleeding disorders community has decades of experience with the infusion of replacement clotting factor and the safety and efficacy are well understood. Non-replacement factor therapies are very different and much more research into their safety and efficacy is required. The management of hemophilia with non-factor replacement therapies is also very different to factor replacement: the principle of “if in doubt, treat” does not apply and must be replaced with an urgent action plan to deal with microbleeds or breakthrough bleeds. The module details how people with hemophilia and their comprehensive care team of healthcare professionals must take into consideration the impact these therapies may have on assays and medical procedures. Illustrations and interactive features make this complex content easy to follow while the glossary and linked resources provide more in-depth explanations. Fun quizzes and progress checks allow the learner to assess their understanding, and point back to specific sections for re-study if necessary.

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