Do we need all that emicizumab?

Abstract

ing protection against bleeds. In both the inhibitor and non-inhibitor patients the remarkable improvement is evidenced in the real-world settings after the extensive clinical phase 3 studies. [1][2][3][4] After stopping the coadministration with activated prothrombin complex concentrate (aPCC, Feiba R ), only a small number of adverse events have been encountered, associated with thrombosis and some breakthrough bleeds. The immediate management option for possible breakthrough bleeds or trauma either with recombinant FVIIa (rFVIIa, Novo Seven R ) or factor VIII concentrate (non-inhibitor patients) should be available for all patients. In our centre we have also advised the patients to immediately contact us (24/7) in case they would need rFVIIa or a replacement therapy.Recent WFH publication reports that prophylactic haemophilia care is not provided globally in 85% of patients. 5 This is mostly related to the

DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with conventional dosing of emicizumab in people with haemophilia A

“…The dose was reduced to result in emicizumab concentrations of ~24 µg/mL, after which the pain resolved and no bleeds occurred during the following 6 months. Subsequently, emicizumab was given in lower doses in 11 PwHA from Finland and 6 PwHA from Thailand without loss of efficacy 19 39. Additionally, real-world evidence from our centre demonstrated similar bleed rates across the concentration subgroups of <40 µg/mL (n=13), 40–80 µg/mL (n=59) and >80 µg/mL (n=22) 15.…”

Emicizumab assays evaluations with four different reagents in severe haemophilia A patients: Concentration from baseline to maintenance therapy

“…7 Moreover, an individualized monitoring of emicizumab concentrations could be useful to optimize the injected dose and allow a more cost-effective dosing. 8 As already described in previously published data, 3 the action of emicizumab leads to a shortened aPTT due to the immediate action of emicizumab in the tenase complex. This effect on aPTT is dose-dependent and observed at low concentration of emicizumab (<13 µg/ml), from the first injection but offers false reassurance because it only reflects a moderate increase in haemostatic capacity.…”

How much prophylaxis is enough in haemophilia?

“…and an average maintenance dose of 1.3 mg/kg/every 2 weeks (43% of recommended) did very well with all, except for one patient, not experiencing any bleeds. 45 The other five published case series were all published in 2023.…”

“…A case series from Finland published in 2022 demonstrated that 11 adult patients (three Inhibitor positive; eight inhibitor negative) receiving an average loading dose of 2.7 mg/kg/wk (90% of recommended) and an average maintenance dose of 1.3 mg/kg/every 2 weeks (43% of recommended) did very well with all, except for one patient, not experiencing any bleeds 45 …”