Abstract Number: OC 08.4
Meeting: ISTH 2021 Congress
Background: Damoctocog alfa pegol (BAY 94-9027, Jivi®) is a site-specific PEGylated recombinant factor VIII (FVIII) molecule with an improved PK profile that proved effective for bleeding protection in patients with hemophilia A treated either twice weekly, every 5 or every 7 days. Head-to-head comparative PK studies showed superiority of damoctocog alfa pegol over efmoroctocog alfa and rurioctocog alfa pegol. WAPPS-Hemo has collected 176 PK studies performed locally at 52 treatment centers worldwide.
Aims: Describe the PK estimates obtained in the real-world setting in patients treated with damoctocog alfa pegol and model the proportion of patients able to achieve pre-defined FVIII troughs according to different treatment regimens.
Methods: The following data were extracted from the WAPPS-Hemo database:
1) Patient demographics;
2) PK estimates;
3) current prophylaxis treatment regimen information.
A modeling exercise was conducted using patient demographics and individual PK profiles to estimate proportion of patients achieving target troughs of interest on commonly used treatment regimens.
Results: One-hundred seventy-six PK studies were retrieved in the database: 160, 15 and 1 performed in severe, moderate and mild patients. The median (range) age at PK assessment was 34.9 years (12-74), body weight 78.5 kg (36-200), dose injected 35.6 IU/kg (8.5-70.5), and dosing interval 3.5 days (1-7). Of the 84 patients with current treatment regimen information available, twice weekly, every 5 days and every 7 days regimens were used in 57%, 7%, and 5% cases, respectively. PK parameters resulted as follows: mean (standard deviation, SD) clearance was 1.73 ml/h/kg (0.60), mean (SD) volume of distribution (Vss) was 36.96 ml/kg (7.43) and mean (SD) terminal half-life was 15.94 hours (4.00). The results of the modeling exercise are reported in the Table.
|N PK studies*||160||160||160||173||173||173||175||175||175|
|N achieving target**||151||92||27||114||38||0||72||14||0|
|% achieving target **||94||56||16||64||21||–||40||8||–|
|Median dose (IU/kg)***||7.7||15.8||31.8||21.3||39.3||–||28.5||47.0||–|
|Max (IU/kg) ***||52.2||58.6||59.3||59.0||59.7||–||58.4||57.0||–|
Conclusions: The analysis of real-world data from WAPPS-Hemo confirms the favourable PK profile shown for damoctocog alfa pegol in the PROTECT VIII study program.
To cite this abstract in AMA style:Mancuso ME, Chelle P, Iserman E, Matthew T, Keepanasseril A, Edginton A, Iorio A, WAPPS-Hemo Investigators . Real-world Use of Damoctocog Alfa Pegol in Patients with Hemophilia A: Analysis of PK Profiles and Treatment Regimen Data from the WAPPS-Hemo Database [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/real-world-use-of-damoctocog-alfa-pegol-in-patients-with-hemophilia-a-analysis-of-pk-profiles-and-treatment-regimen-data-from-the-wapps-hemo-database/. Accessed December 1, 2022.
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