Franchini Massimo, Favaloro Emmanuel J, Lippi Giuseppe
Department of Hematology and Transfusion Medicine, Azienda Ospedaliera Carlo Poma, Mantova, Italy.
Department of Haematology, Institute of Clinical Pathology and Medical Research (ICPMR), Pathology West, Westmead Hospital, Westmead, New South Wales, Australia.
Semin Thromb Hemost. 2015 Oct;41(7):802-8. doi: 10.1055/s-0034-1544004. Epub 2015 Apr 20.
The mainstay of treatment of inherited coagulation disorders is based on the infusion of the deficient clotting factor, when available. Significant advances have been made over the past two decades in the production and availability of factor replacement products. In spite of such progression, several issue are still unsolved, the most important being the need for frequent factor concentrate infusions and the development of inhibitory alloantibodies. To overcome these important limitations, several newer hemostatic agents with an extended half-life are at an advanced stage of clinical development. After a brief overview of hemostasis, this narrative review summarizes the current knowledge on the most promising novel products for hemostasis. The current status of gene therapy for hemophilia, the only therapeutic option to definitively cure this inherited bleeding disorder, is also concisely discussed.
遗传性凝血障碍的主要治疗方法是在有可用的情况下输注缺乏的凝血因子。在过去二十年中，凝血因子替代产品的生产和供应取得了重大进展。尽管有这样的进展，但仍有几个问题尚未解决，其中最重要的是需要频繁输注凝血因子浓缩物以及抑制性同种抗体的产生。为了克服这些重要限制，几种半衰期延长的新型止血剂正处于临床开发的后期阶段。在简要概述止血后，本篇叙述性综述总结了目前关于最有前景的新型止血产品的知识。还简要讨论了血友病基因治疗的现状，这是明确治愈这种遗传性出血性疾病的唯一治疗选择。
