약학회지

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Table. 2.

Table. 2.

Outcomes for concomitant use of emicizumab with ITI

Author Real World Study Inhibitor titer ITI outcome Adverse Events Other Outcomes
Batsuli G et al. (2019)8) O Inhibitor dropped down to <0.6 BU in three patients, and dropped down up to 8.8 BU in other patients. Three, one, three patients achieved successful, partial successful, and failed tolerance, respectively. Nine bleeding events reported in four patients. No thrombotic events reported. Peak FVIII activity and FVIII recovery; median peak fVIII activity was 90% (range 44%-182%), and median fVIII recovery of 45% expected (range 29%-95%)
Carcao M et al. (2019)9) X - - - -
Nagao A et al. (2019)10) O The inhibitor titers tended to decrease in all three cases, - No thrombotic events nor any related laboratory abnormalities were reported. Dosing interval: dosing interval of rFVIII decreased.
Wieland I et al. (2019)11) O Inhibitor decreased from 12.4 BU to <0.6 BU. Successful tolerance in one patient. - IgG subclasses: gG1, IgG3 and 4 increased again with a redominance of IgG4.
Batsuli G et al. (2020)12) O Inhibitor dropped down to <0.6 BU in four patients Three, one, two patients achieved successful, partial successful, and failed tolerance, respectively. One had inhibitor relapse. No thrombotic events reported. -
Camelo RM et al. (2021)13) X - - - Cost-Effectiveness; ITI+EMI is costeffective option compared with ITI+BPA
Chuansumrit A et al. (2021)14) O Inhibitor decreased from 65 BU to 10 BU. Failed tolerance in one patient. Frequent bleeding at the muscles and joints were reported. -
Garcia J et al. (2021)15) O - - No breakthrough bleeding nor TMA/thrombosis were reported. -
Escuriola-Ettingshausen C et al. (2021)16) X - - - Primary endpoint for ITI outcome: inhibitor titer, FVIII recovery
Matsushita T et al. (2022)17) X - - - Assessments: ITI response, safety, and patients and caregiver-reported outcomes.

*BPA: bypassing agent; EMI: emicizumab; ITI: immune tolerance induction; TMA: thrombotic microangiopathy events.;

Yakhak Hoeji 2023;67:75-84 https://doi.org/10.17480/psk.2023.67.2.75
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