Schafer Kim, Munn James, Khair Kate, Thukral Neelam, Tom Angela, McAlister Sally
University of California at Davis, Sacramento, California (Ms Schafer); University of Michigan, Ann Arbor, Michigan (Mr Munn); Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom (Dr Khair); Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana (Ms Thukral); and Biogen, Weston, Massachusetts (Ms Tom and Ms McAlister). Kim Schafer, MSN, RN, is the nurse coordinator at the UC Davis Hemophilia Treatment Center. She has 16 years of experience providing care for both adult and pediatric patients with inherited bleeding disorders. James Munn, MS, BSN is a program and nurse coordinator of the Hemophilia and Coagulation Disorders Program at the University of Michigan Health System. He currently chairs the World Federation of Hemophilia's Nurses Committee and is the treasurer of the Nursing Forum of the International Society on Thrombosis and Haemostasis. Kate Khair, PhD, SRN, RSCN, MSc, MCGI, is a consultant nurse and visiting professor in health and social care at Great Ormond Street Hospital for Children NHS Foundation Trust. She has a particular interest in the quality of life and the lived experience of people with bleeding disorders. Neelam Thukral, CCRC, is a clinical research coordinator at the Indiana Hemophilia & Thrombosis Center, Inc. Angela Tom, MS, BSN, is a medical science liaison at Biogen. She relies on her previous experience as a nurse practitioner in a hemophilia treatment center to inform her scientific engagement with hemophilia providers. She received her BSN and MS from the University of Arizona. Sally McAlister, BSN, has worked in hemophilia for 38 years, including serving as the University of Michigan Hemophilia Treatment Center (HTC)'s nurse coordinator, executive director for the Hemophilia Foundation of Michigan, and director of the HTC Program at the Centers for Disease Control and Prevention (CDC). She currently serves as director, US Medical at Biogen.
J Infus Nurs. 2017 Jan/Feb;40(1):65-75. doi: 10.1097/NAN.0000000000000205.
Prophylaxis for hemophilia A with conventional factor VIII (FVIII) products requires frequent intravenous dosing, which may reduce adherence. Recombinant factor VIII Fc fusion protein (rFVIIIFc) has a prolonged half-life compared with conventional rFVIII, and has demonstrated safety and efficacy for the prevention and treatment of bleeding episodes in phase 3 studies of patients with severe hemophilia A. Most subjects experienced reduced prophylactic dosing frequency with rFVIIIFc compared with prestudy FVIII; the median total weekly prophylactic consumption was comparable. No subjects developed inhibitors. These results suggest that prophylaxis with rFVIIIFc in patients with hemophilia A may allow less frequent prophylactic dosing while maintaining efficacy, with comparable prophylactic consumption.
使用传统的凝血因子 VIII（FVIII）产品对甲型血友病进行预防需要频繁静脉给药，这可能会降低依从性。与传统的重组 FVIII 相比，重组凝血因子 VIII Fc 融合蛋白（rFVIIIFc）的半衰期更长，并且在重度甲型血友病患者的 3 期研究中已证明其在预防和治疗出血发作方面的安全性和有效性。与研究前使用的 FVIII 相比，大多数受试者使用 rFVIIIFc 后的预防性给药频率降低；每周预防性总消耗量中位数相当。没有受试者产生抑制剂。这些结果表明，甲型血友病患者使用 rFVIIIFc 进行预防可能允许减少预防性给药频率，同时保持疗效，且预防性消耗量相当。
