Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy.
Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.
Haemophilia. 2019 Nov;25(6):946-952. doi: 10.1111/hae.13834. Epub 2019 Aug 16.
The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half-life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real-life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half-life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half-life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B.
得益于从标准产品向延长半衰期产品的转变,血友病替代疗法的概念正在发生重大变化。这些新型药物通过减少输注频率、维持更高的谷浓度以确保较低的出血风险,以及通过提高生活质量显著减少对患者的治疗困扰,正在显示出有益的效果。新型药物的疗效及其对血友病 A 和 B 患者常规管理的影响的真实数据仍然有限。本文报告了 2018 年初欧洲血友病和相关疾病协会 (EAHAD) 进行的一项关于使用延长半衰期重组 FVIII 和 FIX 融合产品的患者临床管理的欧洲调查结果,因为在调查时还没有任何聚乙二醇化产品。我们报告了已经转向延长半衰期融合产品的 33 个欧洲血友病中心的这些新型药物的疗效数据,显示出输注次数显著减少,血友病患者的临床治疗中谷浓度令人满意,对血友病 B 的影响更大。