Klukowska A, Laguna P, Svirin P, Shiller E, Vdovin V
Medical Academy Warsaw, Warsaw, Poland.
Haemophilia. 2008 May;14(3):531-8. doi: 10.1111/j.1365-2516.2008.01678.x. Epub 2008 Mar 18.
OCTANINE F is a high-purity blood clotting factor IX concentrate that has been shown to be effective and safe in adults with haemophilia B. At present, there are no prospective clinical study data on FIX replacement therapy in young children. The primary objective of this trial was to investigate the immunogenicity of OCTANINE F in children aged <6 years with haemophilia B. Secondary objectives were to assess the efficacy, viral safety and tolerability of OCTANINE F in this patient population. Twenty-five children aged <6 years with moderate or severe haemophilia B, including six who were previously untreated and 13 who had less than previous 50 exposure days were assigned to prophylactic or on-demand treatment with OCTANINE F over a 12- to 24-month period. Immunogenicity was assessed at baseline, during the treatment period and at the end of treatment by monitoring the levels of inhibitor. OCTANINE F was not associated with the development of an inhibitor in any patient during the study; all patients had a FIX inhibitor level of <0.4 Bethesda units (BU) for all samples taken throughout the study. The efficacy of OCTANINE F was rated as excellent in 96.4% of 499 bleeding episodes and tolerability was rated as very good in 97% of 1684 injections. OCTANINE F was shown to be effective and well tolerated in children aged <6 years with moderate or severe haemophilia B, including previously untreated patients, with no reported cases of FIX inhibitors or thrombotic events.
奥克他宁F是一种高纯度的凝血因子IX浓缩物，已被证明对患有B型血友病的成人有效且安全。目前，尚无关于幼儿FIX替代疗法的前瞻性临床研究数据。本试验的主要目的是研究奥克他宁F在6岁以下B型血友病儿童中的免疫原性。次要目的是评估奥克他宁F在该患者群体中的疗效、病毒安全性和耐受性。25名6岁以下患有中度或重度B型血友病的儿童，包括6名既往未接受治疗的儿童和13名既往暴露天数少于50天的儿童，在12至24个月的时间里被分配接受奥克他宁F的预防性或按需治疗。通过监测抑制剂水平在基线、治疗期间和治疗结束时评估免疫原性。在研究期间，奥克他宁F与任何患者体内抑制剂的产生均无关联；在整个研究过程中采集的所有样本中，所有患者的FIX抑制剂水平均<0.4贝塞斯达单位（BU）。在499次出血事件中，96.4%的事件奥克他宁F的疗效被评为优秀，在1684次注射中，97%的注射耐受性被评为非常好。奥克他宁F在6岁以下患有中度或重度B型血友病的儿童中，包括既往未接受治疗的患者，显示出有效且耐受性良好，未报告FIX抑制剂或血栓形成事件的病例。
