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Ther Adv Hematol. 2018 Feb;9(2):49-61. doi: 10.1177/2040620717746312. Epub 2017 Dec 28.

Novel therapies and current clinical progress in hemophilia A.

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Children's Hospital Los Angeles, Los Angeles, CA, USA.
Children's Hospital Los Angeles, 455 Sunset Boulevard, Mail Stop 54, Los Angeles, CA 90027, USA.


The evolution of hemophilia treatment and care is a fascinating one but has been fraught with many challenges at every turn. Over the last 50 years or so patients with hemophilia and providers have witnessed great advances in the treatment of this disease. With these advances, there has been a dramatic decrease in the mortality and morbidity associated with hemophilia. Even with the remarkable advancements in treatment, however, new and old challenges continue to plague the hemophilia community. The cost of factor replacement and the frequency of infusions, especially in patients with severe hemophilia on prophylaxis, remains a significant challenge for this population. Other challenges include obtaining reliable venous access, especially in younger patients, and the development of neutralizing alloantibodies (inhibitors). The development of extended half-life products, a bispecific antibody which mimics the coagulation function of factor VIII (FVIII) and inhibition of anticoagulation proteins such as antithrombin with antibodies, aptamers or RNA interference technology have offered novel therapeutic approaches to overcome some of these existing challenges. Additionally, ongoing gene therapy research offers a way to possibly cure hemophilia. These novel treatment tools in conjunction with the establishment of an increasing number of comprehensive hemophilia centers and worldwide advocacy efforts have continued to push the progress of hemophilia care to new frontiers. This review highlights and summarizes these novel therapeutic approaches and the current clinical progress of hemophilia A.


anticoagulation inhibition; bispecific antibody; extended half life; gene therapy; hemophilia A; novel therapies

Conflict of interest statement

Conflict of interest statement: Dr Guy Young has the following conflicts of interest: Alnylam, Bayer, Bioverativ, CSL Behring, Genentech, Kedrion, Novo Nordisk, Roche, Shire. Dr Pauline Balkaransingh has none.

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