小儿血友病和出血性疾病治疗的新进展。
New developments in the treatment of pediatric hemophilia and bleeding disorders.
机构信息
Department of Pediatrics, Section of Hematology/Oncology/Bone Marrow Transplantation, Children's Hospital Colorado, University of Colorado - Anschutz Medical Campus, Aurora, Colorado 80045, USA.
出版信息
Curr Opin Pediatr. 2013 Feb;25(1):23-30. doi: 10.1097/MOP.0b013e32835bf77e.
PURPOSE OF REVIEW
Disorders of hemostasis such as hemophilia, von Willebrand disease (VWD), and other clotting protein deficiencies lead to significant morbidity in the pediatric population. Because of the limitations of current treatment options, novel therapies are being developed, many of which are reviewed here.
RECENT FINDINGS
Several new observations about the nature of clotting protein physiology have been made recently, creating novel perspectives on the treatment options. This review will mostly focus on the current therapy as well as new progress in hemophilia care (particularly strategies to prolong half-life of clotting factor replacements, the management of inhibitors, gene therapy, and novel therapeutic approaches), and briefly mention some progress in VWD and fibrinogen deficiency therapies.
SUMMARY
New therapeutic developments have the potential to dramatically decrease morbidity and improve the quality of life of children with bleeding disorders.
目的综述
血友病、血管性血友病 (VWD) 和其他凝血蛋白缺乏等止血障碍会导致儿科人群发病率显著增加。由于目前治疗选择的局限性，正在开发新的治疗方法，其中许多方法在此进行了综述。
最近的发现
最近对凝血蛋白生理学性质的一些新观察结果，为治疗选择创造了新的视角。本综述将主要集中在当前的治疗方法以及血友病治疗方面的新进展（特别是延长凝血因子替代物半衰期的策略、抑制剂的管理、基因治疗和新型治疗方法），并简要提及 VWD 和纤维蛋白原缺乏症治疗方面的一些进展。
总结
新的治疗方法的发展有可能显著降低出血性疾病患儿的发病率并提高其生活质量。
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