ľ¹Ï¸£ÀûÓ°ÊÓ

Hemophilia A is an inherited bleeding disorder in which the body lacks sufficient clotting factor VIII, causing individuals to bleed easily, particularly into the joints, which can lead to permanent damage. Emicizumab (Hemlibra®) is a novel treatment that mimics the function of factor VIII. It is administered under the skin and has a longer duration of action than standard FVIII therapy, reducing the frequency of injections needed.
This thesis consists of four parts. First, we studied over 170 children and found that emicizumab significantly reduced the number of bleeding episodes and made treatment far less burdensome compared to previous therapies. The drug was also shown to be safe, with few side effects.
Next, we investigated whether the thrombin test could reliably indicate emicizumab levels in the blood or predict bleeding risk. The study showed that it could not; results varied greatly within the same patient, making the test unsuitable for monitoring coagulation in patients receiving emicizumab.
The main focus of the thesis was on making emicizumab treatment more efficient and, in the long term, more cost-effective. In two studies, we explored dosage adjustments of emcizumab. By extending the dosing interval to allow full vials to be used, 9% less drug was wasted compared to conventional dosing. The initial results from the nationwide DosEmi study showed that individualized dosing was as effective as the standard regimen, but used nearly 40% less emicizumab.
Finally, we demonstrated that emicizumab may also have potential for use in other rare bleeding disorders.