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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0512 - Real world evidence after switching to turoctocog alfa pegol in patients from Iberian countries

Monday, June 24, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall
Background: Turoctocog alfa pegol (N8-GP) is a glycoPEGylated recombinant factor VIII (FVIII) with an extended half-life developed for treatment in patients with haemophilia A.

Aims: To analyze the clinical impact in patients with Hemophilia A after switching to turoctocog alfa pegol in patients from Iberian countries.

Methods: Observational, retrospective, multicenter study in centers in Spain and Portugal, which includes patients undergoing prophylaxis treatment with turoctocog alfa pegol. It includes as variables: age, frequency of administration and annualized bleeding rates. A comparison is made with the previous treatment of the same patients.

Results: Since February 2020, 95 patients with hemophilia A started prophylaxis with turoctocog alfa pegol. The median follow-up was 32.83 months (range 5.37 – 48.03). The mean age was 34.05 years (range 15-56).
84.21% (80/95) were affected by severe hemophilia A and 84.21% of patients came from a previous prophylaxis regimen
The previous treatment were a standard half-life in 75.78%, extended half-life in 4.21% and plasma-derived FVIII in 20% of patients

Prophylaxis was initiated according to the personalized pharmacokinetic study with the support of WAPPS-hemo in 88.42% (84/95) and with the objective of reaching trough levels of >3-5IU/dL.

The main reason for the change was to reduce the number of infusions in 40% of patients (38/95). Others reasons for the change was: to increase through level, to improve the adherence or to improve clinical parameters

All patients decreased or at least maintained the number of weekly infusions. The mean ABR decreased (2.24 [0-12] vs 0.29 [0-4]).
No complications such as inhibitor development were observed after changing treatment.

Conclusion(s): This study is the largest cohort of patient published in real world life. In this study, turoctocog alfa pegol proved to be an effective therapeutic alternative as a prophylaxis regimen that has allowed us to reduce the treatment burden of patients.