Background: People with hemophilia lack certain proteins (clotting factors) necessary for hemostasis. Its treatment consists in the replacement of these factors. The development of antibodies (inhibitors) that eliminate the administered factor, nullifying its function, is the main therapeutic complication of hemophilia.

Hemophiliacs with inhibitors were usually treated with bypass factors, but we now have new therapeutic resources.

Emicizumab is a bispecific monoclonal antibody with antigen-binding fragments recognizing both FIXa and FX, partially simulating FVIII cofactor role, reconstituting the intrinsic factor Xase reaction despite the presence of FVIII inhibitor antibodies.

Aims: To study the benefits obtained with a careful management strategy of emicizumab simultaneous administration to pediatric patients with severe hemophilia A who developed inhibitors, without neglecting clinical follow-up quality.

Methods: We analyzed retrospective results of in-hospital scheduled emicizumab treatment of 4 children with hemophilia A with inhibitors, over 21 months.

Enrolled children were summoned to come to our Center on the same day (monthly), enabling optimal usage of required vials.

Emicizumab regimen: Loading dose 3mg/kg weekly (4 weeks); maintenance dose 6mg/kg every 4 weeks, for reduced administrations frequency.

Results: In all patients a strict clinical follow-up was confirmed. No bleeding episodes were recorded any patient included in this study. 21 months of simultaneous emicizumab in-hospital administration to our 4 pediatric patients resulted in overall saving of 2520mg, translating into a 25% cost reduction during this period (which, in our case, meant saving 200 133.13€ (116 020.8€ savings per year))

Conclusion(s): Emicizumab therapy requires implementation of a careful management strategy. Our intra-hospital, monthly, simultaneous administrations allowed us to reduce resources consumption, particularly in pediatrics due to significant weight variations, without neglecting appropriate clinical follow-up with more frequent clinical control. Although there are publications recommending shorter intervals administration regimens as more efficient, in our series no bleeding episodes were recorded, thus equaling the effectiveness of intensive regimens.

Table

Table 1: Patients characteristics

Table

Table 2: Total emicizumab consumption during 21 months follow-up: in-hospital administration and predicted home administration

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/emicizumab-treatment-of-4-patients-with-severe-hemophilia-a-and-inhibitors-in-pediatric-age-resource-management-to-minimize-economic-costs-while-maintaining-the-quality-of-clinical-follow-up/