Prophylaxis in adults

The adult secondary prophylaxis study1

Objective

The adult prophylaxis study was the first multicentre, open-label, prospective trial designed to evaluate the effect of secondary prophylaxis, following Factor VIII (FVIII) on-demand treatment, on the number of joint bleeds in adults.

Study criteria

Males (30-45 years old) with severe haemophilia A, negative inhibitor status, history of FVIII treatment (≥100 exposure days), and who were previously treated on-demand. 

  • 16 patients (80%) had ≥1 target joint

Study design

Patients (N=20) received on-demand treatment for 6 months, with a mean dose 31.3 IU/kg per week, followed by 7 months prophylactic treatment (including 1 month washout/stabilisation run-in period, not included in the primary outcome analysis). During prophylaxis, patients received KOGENATE® Bayer 3 times per week; the mean dose was 87.3 IU/kg per week.

Study outcomes

Primary:

  • Number of joint bleeds

Secondary:

  • Number of bleeds
  • Joint function (evaluated by Gilbert score)
  • Health-economics (days off work, general practitioner visits, hospitalisation days)
  • Safety

Results

Joint and total bleeds per patient

Graph - Joint and total bleeds per patient

Patients without target joints (n=4) had 0 bleeds during prophylaxis.

  • The development of more than two target joints increased the risk of bleedings even under prophylaxis treatment

The mean total Gilbert score was significantly better after prophylaxis than on-demand therapy

Mean total Gilbert scores following on-demand and prophylaxis treatment

Graph - Mean total Gilbert scores following on-demand and prophylaxis treatment

  • The recorded decrease in total Gilbert score following prophylaxis was mainly due to the reduced number of bleeds.

No significant difference in health economic parameters (documented at each clinic visit [months 3, 6, 10 and 13]) was noted between on-demand and prophylactic treatment periods.

  • The relatively short observation period of these endpoints may have limited their ability to reflect change
  • The patient population was relatively young and healthy, which may have contributed to the lack of significant differences observed

KOGENATE® Bayer safety data were reassuring

  • Treatment was well tolerated, and no FVIII inhibitor formation was observed

 

Reference:

27. Collins P et al. J Thromb Haemost 2010;8:83-89

UK.PH.HN.KOG.2009.115
January 2010

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Date of preparation: December 2010 UK.PH.HN.KOG.2010.250.30.4