AHAEmi
Brief description of study
The primary objective of the present study is to evaluate the efficacy (effectiveness) of prophylactic (preventative doses) of emicizumab (FDA approved, also called Hemlibra) administered on a regular scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
The secondary objectives are to study the safety of emicizumab in patients with AHA and to evaluate the timing, regimen and effect of immunosuppressive therapy (IST).
Additional objectives- To compare bleeding and adverse events with the historic GTH-AH 01/2010 cohort and to assess the value and risks of time of initiation of IST by comparing with a parallel German/Austrian study. The number of clinically significant bleeds per patient-week until death or week 12 after starting of emicizumab treatment, whatever occurs first. Incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab treatment and mortality in the 24 weeks after starting emicizumab treatment.
Bleeding-free survival in the 12 weeks after starting emicizumab treatment
Mortality and cause of death
Days of treatment with and total dose of bypassing agents (recombinant factor VIIa, activated prothrombin complex concentrate) or recombinant porcine factor VIII (susoctocag alfa) or other factor VIII concentrates
Days in hospital during 12 weeks of emicizumab treatment
Number of patients achieving partial remission in the 24 weeks after starting emicizumab treatment
Specifics (drug, amount and timing) of IST started during the 12 weeks of emicizumab prophylaxis
Tracking PR and CR rates over 12 and 24 weeks
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Medical Research
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Age: - 99 Years
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Gender: All
TBD
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