ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)
ISSN 2383-9457 (ONLINE)
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.;