PERSEPT 11
(PERSEPT = Programme for the Evaluation of Recombinant Factor Seven Efficacy by Prospective Clinical Trial)
PERSEPT 1, a Phase 3 clinical trial, demonstrated the efficacy and tolerability of two CEVENFACTA® initial dose regimens (IDRs) for the treatment of bleeds (severe bleeds were only treated with 225 µg/kg dosing regimen) in subjects with haemophilia A or B with inhibitors.*1,2
BE: bleeding episodes
*Other inclusion criteria: a positive inhibitor test (Bethesda units (BU) ≥5) OR BU titre <5, but expected to have a high anamnestic response to FVIII or FIX OR a BU titre <5, but expected to be refractory to increased dosing of FVIII or FIX
†If an adequate haemostatic response is not achieved e.g. within 24 hours for mild/moderate bleeding episodes, alternative therapies should be considered
‡Severe bleeds were only treated with 225 µg/kg dosing regimen
Global, multicentre, open-label, prospective, randomised, cross-over study of two IDRs in 27 subjects ≥12 years old (range 12–54) with haemophilia A or B with inhibitors who experienced 468 bleeding episodes over the course of the study period.1,2
The successful treatment of a bleeding episode 12 hours after initial administration of CEVENFACTA® and without rebleeding prior to 24 hours
Successful treatment was defined as:
- Bleeds achieving haemostatic efficacy (subject assessment of “Good” or “Excellent” response using a 4-point haemostasis evaluation scale) at 12 hours
- No additional CEVENFACTA® or any alternative treatment prior to 24 hours
- 93% had severe haemophilia, 7% had moderate haemophilia
- Averaged 13 bleeds in the 6 months prior to study entry
- Median 11 bleeds during the study
- Most common joint bleeding episodes were: knee, elbow, ankle/foot and hip
CEVENFACTA® – sustained and reliable control of bleeding with a fast onset of action1
n: number of bleeding episodes; N: number of patients; missing data counted as failures
P-value from one-sided normal approximation test of P, where P is the true proportion of successfully treated mild/moderate bleeding episodes at 12 hours, with adjustment for the correlation among bleeding episodes for a given subject.
Adapted from CEVENFACTA® SmPC1 and Wang et al. 20172
- Median time to attain a “good” or “excellent” assessment for a bleeding episode was 5.98 hours (75 μg/kg IDR) and 3 hours (225 μg/kg IDR)1
- Bleeding control rate 81% (75 μg/kg IDR) and 90.3% (225 μg/kg IDR) at 12 hours1
- Overall bleeding control rate of 96.7% (75 μg/kg IDR) and 99.5% (225 μg/kg IDR) at 24 hours1
CEVENFACTA® was well-tolerated in clinical trials with no thrombotic or thromboembolic events observed1,2,3
PERSEPT 1 (27 subjects, 468 bleeding episodes and 968 injections studied)1,2,3
- 12 subjects reported 25 treatment-emergent adverse events - predominantly non-haemophilia related, mild, and self-resolving2
- 7 treatment-related adverse events (TRAEs) in 2 subjects – all non-serious2,3
- 2 serious adverse events in 1 subject, not treatment-related: tonsillitis and subarachnoid haemorrhage2
Common adverse events (AEs) from Pooled Clinical Trials (75 subjects, 1,117 treatment episodes and 3,418 injections studied):1
Dizziness, headache, injection site discomfort, injection site haematoma, body temperature increased, post-procedural haematoma, injection-related reaction
CEVENFACTA® – Enables patients to get on with their lives
- Median of one (225 μg/kg) and two (75 μg/kg) injections were required to treat a bleeding episode in clinical trials1
- CEVENFACTA® can be easily administered in <2 minutes
References
- CEVENFACTA® Summary of Product Characteristics.
- Wang M, Lawrence JB, Quon DV et al. PERSEPT 1: a phase 3 trial of activated eptacog beta for on-demand treatment of haemophilia inhibitor-related bleeding. Haemophilia 2017;23(6):832–843. doi: 10.1111/hae.13301. Epub 2017 Aug 3. PMID: 28776894.
- Escobar M, Castaman G, Boix SB et al. The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors. Haemophilia 2021;27(6):921–931. doi: 10.1111/hae.14419. Epub 2021 Oct 11. PMID: 34636112.
Further information regarding CEVENFACTA® is available
Additional CEVENFACTA® resources can be found here
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