Manno C S, Butler R B, Cohen A R
Division of Hematology, Children's Hospital of Philadelphia, PA 19104.
J Pediatr. 1992 Nov;121(5 Pt 1):814-8. doi: 10.1016/s0022-3476(05)81921-3.
To assess whether the response and recovery of highly purified factor VIII products approach the levels predicted by use of manufacturers' suggestions, we studied response in vivo and percentage of recovery in boys and men with hemophilia after infusion of 50 IU of an immunoaffinity-purified factor VIII preparation (Hemofil M) per kilogram of body weight. We also studied dose response and percentage of recovery in the same boys after infusion of a factor VII concentrate prepared by solvent detergent treatment and gel filtration (Koate-HP). For boys the difference between mean peak factor VIII response with Hemofil M (1.02% +/- 0.07%) and the response with Koate-HP (1.21% +/- 0.10%) was not significant (p = 0.10), but the response of both products was considerably less than the predicted response of 2.0%. The response to Hemofil M in men (1.38% +/- 0.12%) was significantly better than the response in boys (p = 0.004) but, again, fell short of the anticipated response of 2.0%. In boys the percentage of recovery of Hemofil M was 38.4% +/- 2.1%, compared with Koate-HP recovery, which was 47.0% +/- 3.6% (p = 0.034). The percentage of recovery in men was 47.5% +/- 2.8%. The better response observed in men appears to be, in part, a function of larger body size. We conclude that peak factor VIII response and recovery in boys and men who receive highly purified factor VIII concentrates are lower than those predicted by use of current prescribing recommendations.
为评估高度纯化的凝血因子VIII产品的反应及恢复情况是否接近按照制造商建议所预测的水平，我们研究了血友病男孩和男性在每千克体重输注50国际单位免疫亲和纯化的凝血因子VIII制剂（Hemofil M）后的体内反应及恢复百分比。我们还研究了同一批男孩在输注经溶剂去污剂处理和凝胶过滤制备的凝血因子VII浓缩剂（Koate-HP）后的剂量反应及恢复百分比。对于男孩而言，Hemofil M的平均凝血因子VIII峰值反应（1.02%±0.07%）与Koate-HP的反应（1.21%±0.10%）之间的差异不显著（p = 0.10），但两种产品的反应均明显低于预测的2.0%的反应。男性对Hemofil M的反应（1.38%±0.12%）显著优于男孩（p = 0.004），但同样未达到预期的2.0%的反应。在男孩中，Hemofil M的恢复百分比为38.4%±2.1%，而Koate-HP的恢复百分比为47.0%±3.6%（p = 0.034）。男性的恢复百分比为47.5%±2.8%。在男性中观察到的更好反应似乎部分是由于体型较大。我们得出结论，接受高度纯化凝血因子VIII浓缩剂的男孩和男性的凝血因子VIII峰值反应及恢复低于按照当前处方建议所预测的水平。
