113 Systematic review on PK of emicizumab Figure 3. Emax model with concentration-response relationship of emicizumab in PwHA. Fit of an Emax model of ABRs of treated bleeds according to Ctrough,ss of emicizumab in PwHA (n = 349), described by . Included ABRs were model-based, estimated using negative binominal regression. The EC50 was estimated at 1.47 µg/mL (SE 0.90) and effectiveness plateau was established. Dashed line is 95%-confidence interval and symbol size equals study size. Data from PwHA receiving maintenance were included, see ‘b’ for 11 study subgroups in Supplemental Table ST2. For Ctrough,ss of 30 and 50 µg/mL, sensitivity analyses showed ABRs of treated bleeds of 2.9 and 1.9, respectively, with a conservative setting (baseline ABR 21.9 and Emax 1 [24]) and ABRs of respectively 1.9 and 1.9, respectively, with a more liberal setting (baseline ABR 38.2 and Emax 0.90 [18]), see Supplemental Table ST3. In addition, for ABRs of treated joint bleeds of respectively 1.4 and 0.9 with a conservative setting (baseline ABR 6.7 and Emax 1 [18]) and ABRs of respectively 1.0 and 0.9 with a more liberal setting (baseline ABR 26.5 and Emax 0.97 [19]). ABRbaseline 6
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