Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.
Study Type
OBSERVATIONAL
Enrollment
100
CHU de Caen
Caen, France
Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU
Lille, France
Area under the curve ROC of Residual plasma level of emicizumab
At least one clinically significant bleeding (defined as any bleeding treated with FVIII, rFVIIa or aPCC) from loading period completion (week 5) to the end of study, an average of 1 year
Time frame: At Week 5 (end of emicizumab loading period)
Residual plasma level of emicizumab measured by UPLC-MS/MS
At least one hemarthrosis from loading period completion (week 5) to the end of study, an average of 1 year
Time frame: At Week 5 (end of emicizumab loading period)
Residual plasma level of emicizumab measured by UPLC-MS/MS
Post-traumatic or spontaneous nature of bleeding event
Time frame: At each breakthrough bleeding until end of study
Residual plasma level of emicizumab (UPLC-MS/MS dosing)
Emicizumab FVIII-like activity (chromogenic FVIII BIOPHEN™assay system with emicizumab calibration)
Time frame: At Week 5 and at each breakthrough bleeding until end of study
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