Lund University, Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.
Lancet. 2012 Apr 14;379(9824):1447-56. doi: 10.1016/S0140-6736(11)61139-2. Epub 2012 Mar 27.
Haemophilia care has undergone substantial improvements during the past 40-50 years. Early clotting factor concentrates were not sufficiently refined to enable self-administered treatment at home until the 1970s. Unfortunately, these advances led to transmission of viral diseases including HIV and hepatitis, resulting in an increased burden of morbidity and mortality, especially during the 1980s. Throughout the past two decades, product development, including the advent of recombinant concentrates, has greatly improved the safety and availability of therapy and the focus of care is shifting towards prevention and management of disease sequelae. Long-term substitution therapy (prophylaxis) of the missing clotting factor is the recommended treatment in severe haemophilia, but several research issues remain to be elucidated such as when to start and how to optimise these regimens, and when or whether to stop this expensive treatment. The major side-effect of treatment, development of inhibitors to the infused concentrate, is the main threat to the health of patients and consequently the goal of intense research. Development of new products with improved pharmacokinetics is the next step to improved therapy.
在过去的 40-50 年里，血友病的治疗有了很大的改进。直到 20 世纪 70 年代，早期的凝血因子浓缩物还不够精细，无法在家中进行自我治疗。不幸的是，这些进展导致了包括 HIV 和肝炎在内的病毒疾病的传播，导致发病率和死亡率增加，尤其是在 20 世纪 80 年代。在过去的二十年中，产品开发，包括重组浓缩物的出现，极大地提高了治疗的安全性和可用性，治疗的重点正在转向疾病后遗症的预防和管理。在重度血友病中，缺失凝血因子的长期替代治疗（预防）是推荐的治疗方法，但仍有几个研究问题需要阐明，例如何时开始以及如何优化这些方案，以及何时或是否停止这种昂贵的治疗。治疗的主要副作用，即输注浓缩物产生的抑制剂，是对患者健康的主要威胁，因此也是密集研究的目标。开发具有改善药代动力学的新产品是改善治疗的下一步。
