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目的评估人基因重组FⅧ(rFⅧ-FS)治疗血友病A患者的有效性和安全性。方法2007年10月至11月中国医学科学院血液病医院对16例有急性出血症状血友病A患者进行血液生化检查以及相关病毒免疫学检测,测定rFⅧ-FS治疗前和治疗后1 h以及治疗后12 h的血浆FⅧ:C,检测治疗前后血浆FⅧ抑制物滴度。结果16例患者共给予88次(平均每例患者输注5.5次)rFⅧ-FS输注,共112 089 IU(平均每个患者每次输注1273.7 IU),15例(93.7%)患者输注rFⅧ-FS后出血症状好转,1例(6.3%)患者输注后治疗效果不佳,患者输注后1 h(23.2%±13.5%,P=0.0001)和12 h(12.7%±7.6%,P=0.0001)的FⅧ:C显著高于输注前FⅧ:C(1.1%±1.1%)。所有患者治疗结束后经检测未发现FⅧ抑制物产生。治疗过程中1例患者出现幻视,停药后24 h症状消失。结论rFⅧ-FS在治疗血友病A患者中具有良好的疗效和安全性。
Objective To evaluate the efficacy and safety of human recombinant FⅧ (rFⅧ-FS) in the treatment of hemophilia A patients. Methods From October to November, 2007, hematology hospital of Chinese Academy of Medical Sciences performed blood biochemical examination and related virus immunological tests on 16 patients with hemophilia A with acute hemorrhagic symptoms. The levels of rFVIII-FS before treatment, 1 h after treatment, After 12 h of plasma F Ⅷ: C, plasma FⅧ inhibitor titers were measured before and after treatment. Results Sixteen patients received a total of 88 rFVIII-FS infusions (an average of 5.5 transfusions per patient) for a total of 112,089 IU (1273.7 IU per infusion per patient) and 15 (93.7%) infusions After 1 year (23.2% ± 13.5%, P = 0.0001) and 12 h (12.7% ± 7.6%, P = 0.0001) after the infusion of rFⅧ-FS, P = 0.0001), FⅧ: C was significantly higher than before FⅧ: C (1.1% ± 1.1%). FⅧ inhibitor was not detected in all patients after the treatment. One patient had hallucinations during the course of treatment, and the symptoms disappeared 24 hours after treatment. Conclusions rFⅧ-FS has good curative effect and safety in the treatment of hemophilia A patients.