White G C
Department of Medicine, University of North Carolina, Chapel Hill, USA.
Acta Haematol. 1995;94 Suppl 1:53-7; discussion 57-8. doi: 10.1159/000204031.
Prothrombin complex concentrates (PCCs) have been used for the treatment of hemorrhagic episodes in patients with hemophilia B but have been associated with a high incidence of thrombotic complications. Newer, ultrapure concentrates of factor IX contain less extraneous proteins than PCCs and are less thrombogenic in vitro. In this report, the results of clinical studies with Mononine (Armour Pharmaceutical Company), a purified factor IX concentrate prepared by monoclonal affinity chromatography, are described. Two studies were performed, a phase I/II clinical trial in 10 patients and a compassionate use study in 72 patients. The pharmacokinetics of Mononine were found to be similar to the pharmacokinetics of factor IX after administration of PCCs. In both studies, Mononine was well-tolerated and hemostatically effective when used in doses of up to 161 IU/kg in treatment courses of up to 67 infusions of Mononine. Patients with hepatitis and a prior history of thrombosis with PCCs tolerated Mononine with no evidence of thrombosis. The only thrombotic complication attributed to Mononine in the two studies was an episode of phlebitis at the site of an intravenous line in one patient. These studies indicate that Mononine is safe and effective in the treatment of hemorrhagic episodes in patients with hemophilia B.
凝血酶原复合物浓缩剂（PCCs）已被用于治疗乙型血友病患者的出血发作，但与血栓形成并发症的高发生率相关。较新的超纯IX因子浓缩剂比PCCs含有更少的外来蛋白质，并且在体外血栓形成性较小。在本报告中，描述了使用通过单克隆亲和色谱法制备的纯化IX因子浓缩剂莫罗凝血因子（Armour制药公司）的临床研究结果。进行了两项研究，一项是对10名患者的I/II期临床试验，另一项是对72名患者的同情用药研究。发现莫罗凝血因子的药代动力学与给予PCCs后IX因子的药代动力学相似。在两项研究中，当在高达67次输注莫罗凝血因子的治疗疗程中以高达161 IU/kg的剂量使用时，莫罗凝血因子耐受性良好且具有止血效果。患有肝炎且既往有PCCs血栓形成病史的患者对莫罗凝血因子耐受性良好，没有血栓形成的证据。在两项研究中，唯一归因于莫罗凝血因子的血栓形成并发症是一名患者静脉输液部位的静脉炎发作。这些研究表明，莫罗凝血因子在治疗乙型血友病患者的出血发作方面是安全有效的。
