Background: Emicizumab has been available in Germany for pediatric patients without inhibitors for a year now.We describe five patients who were switched and why.We give details about the Emicizumab levels and our monitoring protocol.

Aims: We want to show which patients were switched, their monitoring and outcome with regard to bleeding and patient satisfaction.

Methods: Five of our patients were switched from FVIII prophylaxis to Emicizumab ,one a PUP, age range 8 months to 15 yrs. Reasons for switching: no venous access ( port implantation mandatory in 3 patients), unwillingness to tolerate infusion via an indwelling port in one case, unwillingness to continue infusion vie a vein or the port in another case.
A further patient cannot infuse himself because he squints. We measure the Emicizumab level in the blood after the loading period before we start the medication every two weeks, I.e. after 6 weeks of treatment and then every 3 m.
Emicizumab levels were all in the desired range, though recommended dose was not implemented due to vial size in some cases. Even a lower dose every two weeks resulted in levels of 60 ng/ ml.
Twice parents gave factor,in one case due to a forehead hematoma. One dose was given after the child had cut himself with scissors.All patients have emergency cards and an emergency supply of FVIII factor at home, one to two doses.
Patient satisfaction and care giver satisfaction is high, though, the smaller children cry briefly when injected.

Results: We report satisfying results with minor bleeding. The PUP who has been treated now for 10 months never had a bleeding event and has not yet needed factor VIII.

Conclusions: We have switched five of our patients and report smooth and easy to implement switching processes with our management protocol.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/real-life-use-of-emicizumab-in-pediatric-patients-without-inhibitors/