Abstract Number: PB0480
Meeting: ISTH 2021 Congress
Background: Emicizumab is a bispecific antibody mimicking the action of coagulation factor VIII, approved for treatment of patients affected by Hemophilia A, without or with inhibitors. Very low concentrations of emicizumab are sufficient to normalize the activated partial thromboplastin time (APTT) in treated patients, thus conventional APTT-based assays are not useful to monitor emicizumab therapy. Here we studied thrombin generation (TG) and fibrin clot formation (FCF) profiles in two adult patients without inhibitors starting emicizumab treatment.
Aims: Our aim was to investigate whether global-coagulation-assays (GCA) could be used to monitor non-factor replacement therapy with emicizumab.
Methods: TG was measured with Calibrated-Automated-Thrombogram and with the automated ST Genesia assays (Stago, Asnières-sur-Seine, France). FCF was measured with the innovative Thrombodynamics Analyzer (LLC Hemacore, Moscow, Russia), which monitors the spatio-temporal (tissue factor-dependent and -independent) dynamics of coagulation. The patients received a weekly dose of Hemlibra® (Hoffmann-La Roche, Switzerland) (3 mg/kg per body weight from week (W) 1 to W4, and 1.5 mg/kg from W5 onwards). Response to treatment was monitored weekly for two months. Analyses of TG and FCF were performed in platelet poor plasma in presence of TF and phospholipids.
Results: In both patients we observed that: i) emicizumab improved TG and FCF compared to baseline; ii) TG normalized after two weeks of treatment; FCF normalized already after one week; iii) both TG and FCF reached a plateau (starting at W4) that lasted until the end of the monitoring. Of note, we observed during emicizumab therapy specific individual coagulation profiles, which correlated with the clinical response.
Conclusions: According to this limited experience, emicizumab seems to improve the hemostatic potential in an individual-specific and “all-or-nothing” manner. TG/FCF assays seem to be promising tools to evaluate the degree of correction exerted by emicizumab on hemostasis, thus possibly personalizing patient treatment. Further investigations are needed to confirm this observation.
To cite this abstract in AMA style:Bertaggia Calderara D, G Zermatten M, Aliotta A, P Batista Mesquita Sauvage A, Alberio L. Usefulness of Global Coagulation Assays to Monitor Emicizumab Therapy [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/usefulness-of-global-coagulation-assays-to-monitor-emicizumab-therapy/. Accessed December 3, 2021.
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