Zanon Ezio
Hemophilia Center, General Internal Medicine, Department of Medicine Padua University Hospital, 35128 Padua, Italy.
Pharmaceuticals (Basel). 2023 Aug 22;16(9):1195. doi: 10.3390/ph16091195.
Hemophilia A is characterized by a deficiency of clotting factor VIII (FVIII) requiring lifelong prophylactic treatment, typically with recombinant FVIII. In recent years, drugs with extended half-lives have become available, including damoctocog alfa pegol (Bayer S.p.A.). The clinical efficacy and safety of damoctocog alfa pegol were demonstrated in the PROTECT VIII phase II/III development program. To assess the physicians' experience and to collect data on prophylactic treatment with damoctocog alfa pegol, a monitoring survey was carried out among 15 Italian hemophilia centers. A total of 149 patients on treatment with damoctocog alfa pegol for at least 6 months were considered. Zero bleeds were reported in 75% of patients treated with damoctocog alfa pegol in the last 6 months; zero hemarthroses were reported in 82% of the same patients. Overall, 86% of patients with damoctocog alfa pegol reduced their monthly infusions. The estimated average reduction in IU/kg during prophylaxis with damoctocog alfa pegol, both monthly and annually, was around 17.5%. All involved clinicians were satisfied with damoctocog alfa pegol. Survey results suggest that damoctocog alfa pegol reduced the number of weekly infusions, annual bleeding, and joint bleeding rate in the majority of patients, improving joint health and patients' quality of life.
甲型血友病的特征是凝血因子VIII(FVIII)缺乏,需要终身预防性治疗,通常使用重组FVIII。近年来,出现了半衰期延长的药物,包括达莫妥珠单抗聚乙二醇(拜耳公司)。达莫妥珠单抗聚乙二醇的临床疗效和安全性在PROTECT VIII II/III期开发项目中得到了证实。为了评估医生的经验并收集有关达莫妥珠单抗聚乙二醇预防性治疗的数据,在15个意大利血友病中心进行了一项监测调查。共有149例接受达莫妥珠单抗聚乙二醇治疗至少6个月的患者被纳入研究。在过去6个月中,75%接受达莫妥珠单抗聚乙二醇治疗的患者报告无出血;同样这些患者中有82%报告无关节积血。总体而言,86%接受达莫妥珠单抗聚乙二醇治疗的患者减少了每月的输注次数。在使用达莫妥珠单抗聚乙二醇进行预防性治疗期间,估计每月和每年每千克体重国际单位的平均减少量约为17.5%。所有参与的临床医生对达莫妥珠单抗聚乙二醇都很满意。调查结果表明,达莫妥珠单抗聚乙二醇减少了大多数患者的每周输注次数、年出血次数和关节出血率,改善了关节健康和患者的生活质量。