Huang James N, Liesner Ri, Austin Steven K, Kavakli Kaan, Akanezi Chioma
UCSF Benioff Children's Hospital and University of California San Francisco San Francisco CA USA.
Haemophilia Comprehensive Care Centre Great Ormond Street Hospital NHS Foundation Trust London UK.
Res Pract Thromb Haemost. 2021 Jul 2;5(5):e12550. doi: 10.1002/rth2.12550. eCollection 2021 Jul.
Coagadex is a high-purity plasma-derived factor X concentrate (pdFX) developed to treat hereditary factor X deficiency (FXD).
Evaluate the efficacy and safety of pdFX administered to patients with hereditary FXD.
This was an open-label, multicenter, retrospective analysis of patients receiving pdFX for compassionate use. Efficacy end points included treatments administered, the number and treatment of bleeds, and investigator assessments. Adverse drug reactions (ADRs) were monitored.
Fifteen patients were included: seven received routine prophylaxis, seven received on-demand treatment, and one alternated. Most were aged ≥12 years (n = 13) and had severe hereditary FXD (n = 12). The median follow-up time was 19.2 months (range, 3.5-48.8). The number of infusions per patient per month was higher for the routine prophylaxis group (median [range], 5.4 [1.4-10.1]) than for the on-demand group (0.8 [0.1-2.3]), as was the dose per infusion (27.9 [21.9-53.6] IU/kg vs 20.0 [13.6-27.7] IU/kg). Patients experienced 88 bleeds (34 minor, 7 major, 47 unclassified). The monthly bleed rate per patient was 0.04 in the routine prophylaxis group (based on 17 bleeds in four patients) and 0.8 in the on-demand group (based on 71 bleeds in eight patients). pdFX was used to treat 79 bleeds and was rated effective in all instances. In an overall assessment, investigators rated pdFX as excellent for 14 patients (93.3%) and good for 1 patient (6.3%). No ADRs or safety concerns were reported.
This analysis supports the use of pdFX as a safe, effective treatment for hereditary FXD. Routine prophylaxis with pdFX may reduce bleed frequency.
Coagadex是一种高纯度的血浆源性X因子浓缩物（pdFX），用于治疗遗传性X因子缺乏症（FXD）。
评估pdFX用于遗传性FXD患者的疗效和安全性。
这是一项对接受pdFX用于同情用药的患者进行的开放标签、多中心回顾性分析。疗效终点包括给药次数、出血次数和治疗情况以及研究者评估。监测药物不良反应（ADR）。
纳入15例患者：7例接受常规预防，7例接受按需治疗，1例交替治疗。大多数患者年龄≥12岁（n = 13），患有严重遗传性FXD（n = 12）。中位随访时间为19.2个月（范围3.5 - 48.8个月）。常规预防组患者每月的输注次数（中位数[范围]，5.4[1.4 - 10.1]）高于按需治疗组（0.8[0.1 - 2.3]），每次输注的剂量也是如此（27.9[21.9 - 53.6]IU/kg对20.0[13.6 - 27.7]IU/kg）。患者发生88次出血（34次轻微出血、7次严重出血、47次未分类出血）。常规预防组患者每月的出血率为0.04（基于4例患者的17次出血），按需治疗组为0.8（基于8例患者的71次出血）。pdFX用于治疗79次出血，所有病例均被评为有效。在总体评估中，研究者将pdFX评为优秀的有14例患者（93.3%），评为良好的有1例患者（6.3%）。未报告ADR或安全问题。
该分析支持将pdFX作为遗传性FXD的安全、有效治疗方法。pdFX常规预防可能会降低出血频率。
