Michigan State University, East Lansing, MI, USA.
Duke University, Durham, NC, USA.
J Thromb Haemost. 2018 May;16(5):849-857. doi: 10.1111/jth.13983. Epub 2018 Apr 10.
Essentials Plasma-derived factor X concentrate (pdFX) is used to treat hereditary factor X deficiency. pdFX pharmacokinetics, safety and efficacy were assessed in factor X-deficient women/girls. Treatment success rate was 98%; only 6 adverse events in 2 subjects were possibly pdFX related. On-demand pdFX 25 IU kg was effective and safe in women/girls with factor X deficiency.
Background A high-purity, plasma-derived factor X concentrate (pdFX) has been approved for the treatment of hereditary FX deficiency, an autosomal recessive disorder. Objective To perform post hoc assessments of pdFX pharmacokinetics, safety and efficacy in women and girls with hereditary FX deficiency. Patients/Methods Subjects aged ≥ 12 years with moderate/severe FX deficiency (plasma FX activity of < 5 IU dL ) received on-demand or preventive pdFX (25 IU kg ) for ≤ 2 years. Results Of 16 enrolled subjects, 10 women and girls (aged 14-58 years [median, 25.5 years]) received 267 pdFX infusions. Mean monthly infusions per subject were higher among women and girls (2.48) than among men and boys (1.62). In women and girls, 132 assessable bleeding episodes (61 heavy menstrual bleeds, 47 joint bleeds, 15 muscle bleeds, and nine other bleeds) were treated with pdFX, with a 98% treatment success rate versus 100% in men and boys. Mean pdFX incremental recovery was similar in the two groups (2.05 IU dL versus 1.91 IU dL per IU kg ), as was the mean half-life (29.3 h versus 29.5 h). Of 142 adverse events in women and girls, headache was the most common (12 events in six subjects). Six events (two infusion-site erythema, two fatigue, one back pain, one infusion-site pain) in two subjects were considered to be possibly pdFX-related. Following the trial, pdFX was used to successfully maintain hemostasis in two subjects undergoing obstetric delivery. Conclusions pdFX was well tolerated and effective in women and girls with FX deficiency. Although women and girls had different bleeding symptoms and sites than men and boys, their pdFX pharmacokinetic profile was comparable.
Essentials 血浆衍生因子 X 浓缩物（pdFX）用于治疗遗传性因子 X 缺乏症。评估了因子 X 缺乏的女性/女孩中 pdFX 的药代动力学、安全性和疗效。治疗成功率为 98%；仅 2 名受试者的 6 起不良事件可能与 pdFX 相关。按需给予 25IUkg 的 pdFX 在因子 X 缺乏的女性/女孩中是有效且安全的。
背景：一种高纯度的血浆衍生因子 X 浓缩物（pdFX）已被批准用于治疗遗传性 FX 缺乏症，这是一种常染色体隐性疾病。目的：对因子 X 缺乏的女性和女孩中 pdFX 的药代动力学、安全性和疗效进行事后评估。
患者/方法：年龄≥12 岁且有中重度 FX 缺乏症（血浆 FX 活性<5IU/dL）的受试者接受按需或预防性 pdFX（25IUkg）治疗，最长 2 年。
16 名入组的受试者中，有 10 名女性和女孩（年龄 14-58 岁[中位数，25.5 岁]）接受了 267 次 pdFX 输注。与男性和男孩相比，女性和女孩的每位受试者每月平均输注次数更高（2.48 次比 1.62 次）。在女性和女孩中，132 次可评估的出血事件（61 次重度月经出血、47 次关节出血、15 次肌肉出血和 9 次其他出血）用 pdFX 治疗，成功率为 98%，男性和男孩的成功率为 100%。两组的 pdFX 增量恢复相似（每 IUkg 增加 2.05IU/dL 比 1.91IU/dL），半衰期也相似（29.3h 比 29.5h）。在女性和女孩的 142 次不良事件中，头痛最常见（6 名受试者共 12 次）。两名受试者的 6 次事件（两次输注部位红斑、两次疲劳、一次背痛、一次输注部位疼痛）被认为可能与 pdFX 相关。试验后，两名接受产科分娩的受试者使用 pdFX 成功维持止血。
pdFX 在 FX 缺乏的女性和女孩中耐受性良好且有效。尽管女性和女孩的出血症状和部位与男性和男孩不同，但她们的 pdFX 药代动力学特征相似。
