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Review
. 2016 Dec 2;2016(1):650-656.
doi: 10.1182/asheducation-2016.1.650.

New therapies for hemophilia

Steven W Pipe  1
Affiliations
Review

New therapies for hemophilia

Steven W Pipe. Hematology Am Soc Hematol Educ Program. .

Abstract

Individuals with severe hemophilia have benefitted from 5 decades of advances that have led to widespread availability of safe and efficacious factors VIII and IX, a multidisciplinary integrated care model through a network of specialized hemophilia treatment centers, and aggressive introduction of prophylactic replacement therapy to prevent bleeding and preserve joint health. Yet, there are remaining challenges and treatment gaps which have prevented complete abrogation of all joint bleeding, and progressive joint deterioration may continue in some affected individuals over the course of a lifetime. Some of these challenges can now be addressed with recombinant clotting factors with extended half-life that may improve adherence to prophylaxis regimens through more convenient infusion schedules, maintain higher plasma levels for longer when clinically necessary, and allow for better adaptation to individual phenotypic and pharmacokinetic variability. Real-world case studies will be presented that illustrate practical application of these newly approved therapies in clinical practice and the clinical trial data that have demonstrated the potential for improved clinical outcomes by implementing these strategies.

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Conflict of interest statement

Conflict-of-interest disclosure: S.W.P. is on the board of directors or an advisory committee for the American Thrombosis and Hemostasis Network, the Medical and Scientific Advisory Council for the National Hemophilia Foundation and has consulted for Baxalta, Novo Nordisk, CSL Behring, Biogen, Bayer, and Genentech/Roche.

Figures

Figure 1.
Figure 1.
Major advances in therapy for hemophilia and their associated outcomes.
Figure 2.
Figure 2.
Current outcomes and remaining challenges in therapeutic strategies for hemophilia.
Figure 3.
Figure 3.
FVIII and FIX therapeutic agents recently approved or in late-phase clinical development. Approved agents are presented with their trademarked names as TM. Agents in the late-phase trials discussed in this review are indicated by their clinical names. Fc fusion and albumin fusion denoted by Fc and FP. Human-cl rh indicates human cell line recombinant human. EHL, extended half-life. Adapted from Impact Education, LLC/National Hemophilia Foundation/Postgraduate Institute for Medicine with permission.
Figure 4.
Figure 4.
Strategies to extend the half-life of recombinant clotting factors.
See this image and copyright information in PMC

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