a Department of Internal Medicine and Pediatrics, Division of Hematology , University of Texas Health Science Center and the Gulf States Hemophilia and Thrombophilia Center , Houston , TX , USA.
b Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico , Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre , Milan , Italy.
Expert Rev Hematol. 2019;12(sup1):1-13. doi: 10.1080/17474086.2018.1564032.
: Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia, but this often requires frequent infusions. A number of long-acting factor products have been developed to reduce the burden on patients. : This is an overview of information presented at two symposia held at the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis - Scientific and Standardization Committee annual meetings. The pharmacokinetic, safety and efficacy data for long-acting recombinant products are reviewed, with a focus on recombinant factor IX albumin fusion protein (rIX-FP) and rVIII-SingleChain. This overview also provides a guide for managing a patient's switch to long-acting products. : Long-acting products may allow patients to maintain or decrease bleeding rates whilst increasing their dosing interval, which may in turn reduce the burden on patients and caregivers. When switching patients to long-acting products health-care professionals should provide balanced and thorough education to the patient, whilst supporting their emotional well-being. Regimens should address patients' needs and goals but should also be guided by clinical phenotype and pharmacokinetic assessment. Follow-up should assess safety concerns, bleeding rates, joint health and the impact of the regimen on patients' lifestyle.
预防因子替代疗法是治疗血友病的金标准，但这通常需要频繁输注。已经开发出许多长效因子产品来减轻患者的负担。这是在世界血友病联盟和国际血栓形成与止血学会科学和标准化委员会年会上举行的两个专题讨论会介绍的信息概述。本文综述了长效重组产品的药代动力学、安全性和疗效数据，重点介绍了重组因子 IX 白蛋白融合蛋白(rIX-FP)和 rVIII-SingleChain。该综述还为管理患者向长效产品的转换提供了指导。长效产品可能使患者在维持或减少出血率的同时增加其给药间隔，这反过来可能减轻患者和护理人员的负担。当将患者转换为长效产品时，医疗保健专业人员应向患者提供平衡和全面的教育，同时支持他们的情绪健康。方案应满足患者的需求和目标，但也应根据临床表型和药代动力学评估进行指导。随访应评估安全性问题、出血率、关节健康以及方案对患者生活方式的影响。
