Zentrum für Pädiatrische Hämostaseologie, Ludwig-Maximilian Universität, Klinikum der Universität, Campus Innenstadt, Lindwurmstraße 4, 80337 München, Germany.
Prof. Hess Kinderklinik, Klinikum Bremen Mitte, Bremen, Germany.
Thromb Res. 2014 Nov;134 Suppl 1:S27-32. doi: 10.1016/j.thromres.2013.10.017. Epub 2013 Nov 17.
Prophylaxis is now an established treatment standard in haemophilia in Western Europe and the US with multiple studies demonstrating the clinical benefits of prophylaxis over on-demand treatment. In Western Europe in particular, prophylactic use of factor VIII (FVIII) is high as a result of the findings from the early prophylaxis studies and adherence to national guidelines. Unfortunately, prophylaxis has not yet been implemented on a worldwide basis. The introduction of prophylaxis by haemophilia treatment centres in Bremen, Frankfurt and Munich, as recommended in German guidelines, has significantly improved outcomes for our young haemophilia patients. In the Frankfurt centre, a decreasing rate of inhibitors has been observed since prophylaxis was started early, dosing was individualized, and the importance of treatment continuity was recognized. The centres in Munich and Bremen have explored the possibility of further reducing inhibitor rates using early tolerization - a new prophylaxis regimen that introduces low FVIII doses administered once weekly as soon as a bleeding tendency is observed - with excellent results. All three centres avert the induction of immunological danger signals by avoiding the use of central venous catheters, postponing vaccination wherever possible and not undertaking elective surgery during the early FVIII exposure days. The benefits of using this approach have been confirmed by the remarkably low rates of inhibitors in previously untreated patients reported at these centres. Hopefully, as we and others explore new prophylaxis regimens for our paediatric patients, we can work towards the goal of one day overcoming this serious complication of haemophilia treatment.
预防治疗目前已在西欧和美国成为血友病的既定治疗标准，多项研究表明预防治疗相较于按需治疗具有显著的临床优势。在西欧，由于早期预防治疗研究的结果和对国家指南的遵循，VIII 因子（FVIII）的预防性使用非常普遍。遗憾的是，预防治疗尚未在全球范围内实施。按照德国指南的建议，不来梅、法兰克福和慕尼黑的血友病治疗中心开始推行预防治疗，这显著改善了我们年轻血友病患者的预后。在法兰克福中心，自早期开始预防治疗、个体化剂量调整以及认识到治疗连续性的重要性以来，抑制剂的发生率一直在下降。慕尼黑和不来梅中心探索了通过早期耐受化（一种新的预防治疗方案，一旦出现出血倾向，每周一次给予低剂量 FVIII）进一步降低抑制剂发生率的可能性，取得了极好的效果。所有三个中心都通过避免使用中心静脉导管、尽可能推迟疫苗接种以及不在 FVIII 早期暴露期间进行择期手术，避免了免疫危险信号的诱导。这些中心报告的既往未接受治疗患者中抑制剂发生率极低，证实了这种方法的益处。希望随着我们和其他人探索新的儿科预防治疗方案，我们能够朝着有朝一日克服血友病治疗这一严重并发症的目标迈进。
