Inhibitors - PUPs

KOGENATE® Bayer – the evidence on inhibitor formation

Kreuz et al have shown that KOGENATE® Bayer has the lowest published incidence of inhibitors in PUPs and minimally treated patients (MTPs). 1

Objective

To evaluate the safety and efficacy of KOGENATE® Bayer in 61 PUPs. 1

Study design

  • An open-label, non-controlled, multi-centre trial 1
  • Patients were aged four years or younger with severe haemophilia A 1
  • All enrolled patients were on prophylactic or on-demand regimens and treated solely with KOGENATE® Bayer for ≥2 years 1

Primary outcomes

  • The number of adverse events, especially events associated with the infusion of KOGENATE® Bayer. A positive inhibitor titre was considered to be ≥0.6BU 1
  • The number of infusions required to achieve haemostasis for each new bleeding episode 1

Background

  • 21% of patients had a family history of FVIII inhibitor formation 1
  • Patients were of different racial/ethnic backgrounds (48 Caucasian, 5 Black, 8 Other) 1
  • Over half of the patients had Intron 22 inversions or large deletions of the FVIII gene 1
  • The distribution of mutation types in this study population was typical for a population of patients with severe haemophilia 13

Results

  • 15% of patients treated with KOGENATE® Bayer developed inhibitors (n=60) 1
  • Inhibitor formation was transient in two patients with low-titre inhibitor antibodies 1 *

*Transient was defined as "no further positive inhibitor results observed during the subsequent 2 years,
despite continued on-demand treatment with KOGENATE® Bayer"

 

Inhibitor incidences from prospective studies of PUPs with haemophilia A

Data from non-comparative studies. No published head-to-head studies of KOGENATE® Bayer vs ReFacto® exist.

 

KOGENATE® Bayer

Lowest published rate of inhibitor formation in PUPs in non-comparative studies - (15%) 1,2

References

1. Kreuz W et al Thromb Haemost 2005;93:457-467
2. Courter SG and Bedrosian CL. Semin Hematol 2001;38(2)(Suppl 4):52-59
13. Becker J et al. Am J Hum Genet1996;58(4):657-670
29. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-
_Product_Information/human/000520/WC500022467.pdf (Last accessed December 1, 2010)

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Date of preparation: December 2010 UK.PH.HN.KOG.2010.250.19.6