Abstract Number: PB0603
Meeting: ISTH 2021 Congress
Background: Emizicumab has been licensed in Germany for non inhibitor patients for two years now. We have treated the first PUP in Germany, he has had no treated bleed now for two years, we report on further 11 patients with two more PUPs ( age range 4 months to 15 yrs).
Aims: We describe the successful use in a broad range of pediatric patients with severe hemophilia A including 3 PUPs and with varying challenges. We want to point out real life concerns in the treatment regarding compliance , difficulties in dosing due to the limited vial size and in the necessity of additional factor dosing in case of non severe bleeding.
Methods: We describe 12 pediatric patients with the dosing schemes, extended dosing intervals due to vial size, the subsequent Emicizumab levels, the bleeding events and when we chose to add factor, and the overall satisfaction of the patients and their families.
Results: We report the successful outcomes in the treatment with Emicizumab. We report 2 port removals with additional factor ,one dose given in case of head trauma and one traumatic muscle bleed needing additional factor.
The levels taken after adjusted dosing ( every 3 weeks, every ten days e.g.) according to vial size were in the desired range.
Conclusions: We advocate the use of Emicizumab especially in PUPs. Our oldest PUP has been treated now for 2 years thus saving himself 312 iv injections, the the other two PUPs would have needed ports. We have hardly seen bleeds needing factor, we advocate a personalized dosing scheme due to vial size. Questions remain regarding when to treat a bleed and when to wait, whether to try to tolerize a PUP. A very important issue is the complete lack of educational material for families as all the books available do not include Emizicumab.
To cite this abstract in AMA style:Heine S, Graf N. Update on Real Life Use of Emicizumsb in Pediatric Patients without Inhibitors [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/update-on-real-life-use-of-emicizumsb-in-pediatric-patients-without-inhibitors/. Accessed February 27, 2024.
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