Emicizumab: Review of the literature and critical appraisal.
INTRODUCTION
Emicizumab-kywh (ACE910) is a recombinant, humanized, asymmetric bispecific antibody that functions to bring activated FIX (FIXa) and zymogen FX into an appropriate steric conformation to medicate the activation of FX to FXa thereby mimicking the cofactor function of FVIIIa.


AIM
The objective of this manuscript was to review the development and potential role for emicizumab in the treatment of patients with haemophilia A with and without inhibitors.


METHODS
A Cochrane Library and PubMed (MEDLINE) search focusing on emicizumab in haemophilia was conducted.


RESULTS
In total, 37 citations were retrieved and serve as the database for the literature reviewed herein. Once-weekly subcutaneous injection of emicizumab at three dose levels has been shown to be effective as prophylaxis to prevent bleeding in a majority haemophilia A patients with inhibitors to FVIII. Likewise, prevention of bleeding was also observed in more than two thirds of patients without inhibitors to FVIII. One antidrug antibody to emicizumab has been reported in over 600 treated patients, two have developed thromboembolic events and three thrombotic microangiopathy. These thrombotic complications have occurred in conjunction with FVIII-bypassing agents, and none have been observed following recommendations from the manufacturer regarding concomitant use of bypassing agents. The median annual treated bleeding rates were decreased in patients with as well as those without an inhibitor to FVIII.


CONCLUSION
The principal advantage of emicizumab is subcutaneous administration and effectiveness irrespective of the presence of inhibitors. Emicizumab could conceivably represent a new epoch in the treatment of people with haemophilia A.
['Rodriguez-Merchan|E Carlos|EC|http://orcid.org/0000-0002-6360-0113', 'Valentino|Leonard A|LA|http://orcid.org/0000-0003-0420-8565']
D000818:Animals D018033:Antibodies, Bispecific D061067:Antibodies, Monoclonal, Humanized D002986:Clinical Trials as Topic D016208:Databases, Factual D004195:Disease Models, Animal D004305:Dose-Response Relationship, Drug D006207:Half-Life D006261:Headache D002836:Hemophilia B D006801:Humans
emicizumab haemophilia A prophylaxis efficacy safety