Zdziarska Joanna, Chojnowski Krzysztof, Klukowska Anna, Łetowska Magdalena, Mital Andrzej, Podolak-Dawidziak Maria, Windyga Jerzy, Zawilska Krystyna
Department of Hematology, Jagiellonian University School of Medicine, Kraków, Poland.
Pol Arch Med Wewn. 2009 Jun;119(6):403-9.
Advances in hemophilia management in the 20th century enabled effective and early treatment of joint and other bleeds typical of this disease, also in a home setting. Prophylaxis became available as the optimal approach to prevent hemophilic arthropathy and improve patients' quality of life. To increase treatment safety, lyophilized plasma-derived factor VIII and IX concentrates were subjected to numerous procedures designed to decrease the risk of transmission of known and unknown pathogens. During the following years, recombinant factor VIII and factor IX preparations were developed to completely eliminate the risk. Recombinant factor concentrates were extensively studied in terms of their therapeutic properties, safety, and immunogenicity. This article reviews the current knowledge on efficacy and safety of recombinant factors VIII and IX.
20世纪血友病治疗取得的进展使得针对该疾病典型的关节及其他出血情况能够进行有效且早期的治疗,即便在家中也可进行。预防性治疗成为预防血友病性关节病及改善患者生活质量的最佳方法。为提高治疗安全性,冻干血浆源性凝血因子VIII和IX浓缩物经过了多项旨在降低已知和未知病原体传播风险的程序。在随后的几年里,开发出了重组凝血因子VIII和IX制剂以完全消除这种风险。对重组凝血因子浓缩物的治疗特性、安全性和免疫原性进行了广泛研究。本文综述了关于重组凝血因子VIII和IX疗效及安全性的现有知识。