Ljung Rolf
Department of Clinical Sciences Lund-Paediatrics, Lund University, Lund, Sweden ; Department of Paediatrics and Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.
Thromb J. 2016 Oct 4;14(Suppl 1):30. doi: 10.1186/s12959-016-0103-3. eCollection 2016.
Retrospective and prospective studies unambiguously show that prophylactic treatment of severe hemophilia A or B should be started as primary prophylaxis at 1-2 years' of age and ideally before the first joint bleed. The dose and dose frequency should be individually tailored depending on the goal of treatment, venous access and the bleeding phenotype. The circumstances during the first exposures of factor VIII concentrates in hemophilia A may have an impact on the risk of developing inhibitors. Enhanced half-life products, in particular in hemophilia B, will facilitate treatment in patients with difficult venous access but also in achieving a higher trough level. Evidence accumulate that prophylactic treatment is beneficial also in adults and in patients with inhibitors.
回顾性和前瞻性研究明确表明,重度甲型或乙型血友病的预防性治疗应在1至2岁时作为初级预防开始,理想情况是在首次关节出血之前。剂量和给药频率应根据治疗目标、静脉通路和出血表型进行个体化调整。甲型血友病患者首次接触凝血因子VIII浓缩物时的情况可能会对产生抑制剂的风险产生影响。半衰期延长的产品,特别是在乙型血友病中,将有助于静脉通路困难的患者进行治疗,也有助于达到更高的谷浓度。越来越多的证据表明,预防性治疗对成人和有抑制剂的患者也有益。