Prof. Dr. C. T. Nicolau National Institute for Transfusional Haematology, Bucharest, Romania.
Department of Haematology, Ege University Children's Hospital, Izmir, Turkey.
Haemophilia. 2018 Jul;24(4):604-610. doi: 10.1111/hae.13466. Epub 2018 Mar 26.
The pharmacokinetics (PK), efficacy and safety of moroctocog alfa (AF-CC) have been demonstrated in haemophilia A patients aged ≥6 years.
These studies aimed to further describe moroctocog alfa (AF-CC) experience in paediatric patients (<12 years) with severe haemophilia A (FVIII:C < 1%).
Two prospective, open-label studies enrolled patients aged <12 years: one study with 37 previously treated patients (PTPs) and another with 23 previously untreated patients (PUPs). All patients initially received 50 IU/kg of moroctocog alfa (AF-CC) to evaluate either recovery alone, or with other PK parameters (6 to <12 years) before continuing treatment for 100 exposure days (EDs) or 24 months.
At baseline, mean (±SD) recovery ranged between 1.32 ± 0.65 (PUPs aged <2 years) and 2.13 ± 0.82 (PTPs aged 6 to <12 years). The mean (±SD) half-life was 9.12 ± 1.94 hours in PTPs aged 6 to <12 years. No new safety signals were detected in either study, 2 transient lower titre inhibitors occurred in PTPs while 8 inhibitors (3 low and 5 high titre) were detected in PUPs. Most bleeding episodes resolved with one infusion (94% [893/954]). The annualised bleeding rate (ABR) in the PTP study was 27.5 and 4.2 for patients reporting an on-demand and routine prophylaxis regimen at baseline, respectively. In the PUP study, the overall ABR was 5.9.
Moroctocog alfa (AF-CC) had expected PK findings (lower recovery in young children compared with older children) along with being safe and efficacious in a population of young severe haemophilia A patients.
莫罗考托戈阿尔法（AF-CC）在年龄≥6 岁的血友病 A 患者中的药代动力学（PK）、疗效和安全性已得到证实。
这些研究旨在进一步描述莫罗考托戈阿尔法（AF-CC）在年龄<12 岁的严重血友病 A（FVIII：C<1%）患儿中的经验。
两项前瞻性、开放性研究纳入了年龄<12 岁的患者：一项研究纳入了 37 名既往接受过治疗的患者（PTP），另一项研究纳入了 23 名既往未接受过治疗的患者（PUP）。所有患者最初均接受 50IU/kg 的莫罗考托戈阿尔法（AF-CC），以评估恢复情况，或评估 6 至<12 岁患者的其他 PK 参数，然后继续治疗 100 个暴露日（ED）或 24 个月。
在基线时，恢复率的平均值（±SD）范围为 1.32±0.65（年龄<2 岁的 PUP）至 2.13±0.82（年龄 6 至<12 岁的 PTP）。年龄 6 至<12 岁的 PTP 患者的平均（±SD）半衰期为 9.12±1.94 小时。两项研究均未发现新的安全性信号，2 名 PTP 患者出现一过性低滴度抑制剂，8 名 PUP 患者出现 3 种低滴度和 5 种高滴度抑制剂。大多数出血事件单次输注即可解决（94%[893/954]）。PTP 研究中，基线时报告按需和常规预防方案的患者年化出血率（ABR）分别为 27.5 和 4.2。在 PUP 研究中，总体 ABR 为 5.9。
莫罗考托戈阿尔法（AF-CC）在年轻严重血友病 A 患者中具有预期的 PK 特征（与年龄较大的儿童相比，年幼儿童的恢复较低），且安全有效。
