|
Randomized, prospective clinical trial of recombinant factor VIIa
for secondary prophylaxis in hemophilia patients with inhibitors
Summary. Background: Hemophilic patients with factor VIII
(FVIII) and FIX inhibitors suffer from frequent bleeding
episodes and reduced quality of life. Objectives: To evaluate
whether secondary prophylaxis with activated recombinant
factor VII (rFVIIa) can safely and effectively reduce bleeding
frequency as compared to conventional on-demand therapy.
Methods: Thirty-eight male patients entered a 3-month
preprophylaxis period to confirm high baseline bleeding
frequency (mean ‡ 4 bleeds per month). Twenty-two patients
were randomized 1:1 to receive daily rFVIIa prophylaxis with
either 90 or 270 lg kg)1 for 3 months, followed by a 3-month
postprophylaxis period. Results: Bleeding frequency was reduced
by 45% and 59% during prophylaxis with 90 and
270 lg kg)1, respectively (P < 0.0001); however, there was no
significant difference detected between doses. The majority of
this reduction was maintained during the postprophylaxis
period. Although all types of bleed were similarly reduced, the
effect was most pronounced for spontaneous joint bleeds.
Patients reported significantly fewer hospital admissions and
days absent from work/school during prophylaxis as compared
to the preprophylaxis period. No thromboembolic events were
reported during prophylaxis. Conclusion: Clinically relevant
reductions in bleeding frequency during prophylaxis as compared
to conventional on-demand therapy were achieved
without raising safety concerns. These results provide evidence
for the concept of secondary rFVIIa prophylaxis in inhibitor
patients with frequent bleeds.
点击下载全文：抑制物产生的二级预防
|
