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Does Pharmacokinetic-guided Prophylaxis with Antihemophilic Factor (Recombinant) Improve Bleeding Rates over Standard Prophylaxis? Real-world Data from the AHEAD German Study

R. Klamroth1, A. Huth-Kühne2, K. Kurnik3, C. Escuriola-Ettingshausen4, S. Regensburger5, L. Tang6, J. Norton7, A. Fernandez6, J. Oldenburg8

1Department for Internal Medicine, Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Germany, 2SRH Kurpfalzkrankenhaus and Haemophilia Centre Heidelberg GmbH, Heidelberg, Germany, 3Dr. von Haunersches Kinderspital Klinikum der Universität München, Munich, Germany, 4Hämophilie-Zentrum Rhein Main GmbH, Mörfelden-Walldorf, Germany, 5Takeda Pharma Vertrieb GmbH & Co. KG, Berlin, Germany, 6Takeda Pharmaceuticals International AG, Zürich, Switzerland, 7Takeda Development Center Americas, Inc., Lexington, United States, 8Institute for Experimental Hematology and Transfusion Medicine, Bonn University Clinic, Bonn, Germany

Abstract Number: PB0509

Meeting: ISTH 2021 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: The German Antihemophilic factor (recombinant) (rAHF) Hemophilia A (HA) outcome Database (AHEAD) study (DRKS00000556) is a non-interventional prospective multicenter real-world study that assesses long-term effectiveness and safety of rAHF in patients with HA.

Aims: To describe hemostatic effectiveness of rAHF administered as standard prophylaxis (SP) and individualized pharmacokinetic-guided prophylaxis (PKP) in moderate or severe HA.

Methods: The 7th interim analysis describes results from the safety analysis set (SAS) (data cutoff: Jun 30, 2020). Patients were followed over 7 years; 4 years of follow-up are available for SP versus PKP, because day 1 was reset after patients adopted PKP. Key outcomes included annualized bleeding rates (ABRs [all bleeds]), annualized joint bleeding rates (AJBRs), rAHF consumption, factor VIII (FVIII) trough levels, and adverse events (AEs). Ethics committee approval and patients’ informed consent were obtained.

Results: Of 322 patients in SAS (severe HA, n=258; moderate HA, n=64), 250 received prophylaxis (SP and PKP), 67 on-demand treatment, and 5 immune tolerance induction. Median ABRs were lower in patients receiving PKP versus SP over years 1-3. Similar median AJBRs were observed between groups, although the range was wider in patients receiving SP. A higher proportion of patients receiving PKP versus SP had an ABR or AJBR of zero over years 1-3 (Table 1). Mean annualized total dose over years 1-4 was 5260-5437 IU/kg for SP and 4545-5769 IU/kg for PKP. Mean (median [Q1-Q3]) target FVIII troughs for patients with moderate HA at years 1 (n=2) and 2 (n=1) were 2.2 (2.2[1.0-3.4]) and 1.0 (1.0[NA]). For severe HA, mean (median [Q1-Q3]) FVIII troughs for years 1 (n=14) and 2 (n=7) were 4.4 (3.5[2-5]) and 3.4 (3.0[1.5-5.5]). Treatment-related AEs occurred in 31/322 (9.6%) patients; serious treatment-related AEs, 23/322 (7.1%).

Year 1 Year 2 Year 3 Year 4
Bleeding rates SP
n=246
PKP†
n=24
SP
n=229
PKP†
n=12
SP
n=211
PKP†
n=3
SP
n=190
PKP†
n=1
Median ABR
(Q1-Q3)
2 (0-6) 0 (0-2) 2 (0-4) 0 (0-0.5) 1 (0-5) 0 (0-1) 1 (0-4) 3 (3-3)
Median AJBR
(Q1-Q3)
0 (0-2) 0 (0-0.5) 0 (0-2) 0 (0-0) 0 (0-2) 0 (0-1) 0 (0-2) 1 (1-1)
% patients with ABR=0 36 63 36 75 37 67 48 0
% patients with AJBR=0 58 75 59 83 56 67 64 0
Consumption SP
n=246
PKP*
n=24
SP
n=232
PKP*
n=14
SP
n=215
PKP*
n=3
SP
n=195
PKP*
n=1
Mean (SD) annualized total dose, IU/kg 5340
(3254)
5652
(2841)
5437
(3250)
5000
(2004)
5318
(3224)
4545
(941)
5260
(3045)
5769
(NA)
Median (Q1-Q3) annualized total dose, IU/kg 4737
(3143-6789)
4986
(3601-7125)
4805
(3283-6592)
4827
(3900-5778)
4727
(3033-6556)
4111
(3900-5625)
4692
(3033-6592)
5769
(NA)
*All patients receiving prophylaxis started on SP but could be switched to PKP.
†Patients with HA using the FDA-approved mobile app that allows patients to track treatment with rAHF, view estimated factor VIII levels, and share data with healthcare provider.
NA, not available; Q, quartile.

Bleeding Rates and Consumption of rAHF in Patients Receiving SP Versus PKP*

Conclusions: These real-world data show improved bleeding rates with PKP versus SP with similar rAHF consumption.

To cite this abstract in AMA style:

Klamroth R, Huth-Kühne A, Kurnik K, Escuriola-Ettingshausen C, Regensburger S, Tang L, Norton J, Fernandez A, Oldenburg J. Does Pharmacokinetic-guided Prophylaxis with Antihemophilic Factor (Recombinant) Improve Bleeding Rates over Standard Prophylaxis? Real-world Data from the AHEAD German Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/does-pharmacokinetic-guided-prophylaxis-with-antihemophilic-factor-recombinant-improve-bleeding-rates-over-standard-prophylaxis-real-world-data-from-the-ahead-german-study/. Accessed September 24, 2023.

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