Scott J P, Montgomery R R
Department of Pediatrics, Medical College of Wisconsin, Milwaukee.
Semin Thromb Hemost. 1993;19(1):37-47. doi: 10.1055/s-2007-994004.
Impressive progress has been made in the treatment of von Willebrand disease. Desmopressin is the drug of choice for mild to moderate type I von Willebrand disease patients in whom adequate hemostatic levels of vWF activity can be achieved. Cryoprecipitate from random donors is no longer an optimal choice because of the risk of transfusion-associated viral infections. In special circumstances, cryoprecipitate from a very small donor pool, particularly if obtained following desmopressin stimulation, remains an attractive alternative because these donors can be intensively screened. This therapy may also be somewhat less expensive than commercial concentrates. Virally attenuated commercial concentrates containing intact vWF multimers are currently the treatment of choice to achieve high levels of vWF for moderate to severe von Willebrand disease and for patients with variants of von Willebrand disease that cannot be adequately treated with desmopressin or for whom desmopressin is contraindicated. It is hoped that concentrates of vWF specifically designed for treatment of von Willebrand disease will prove to be safe and efficacious. Standardized assays of vWF in concentrates need to be established. Although, the optimal treatment product has not been produced, several of the more recently developed products have structures that more closely resemble intact normal plasma vWF and appear promising.
血管性血友病的治疗已取得显著进展。去氨加压素是治疗轻至中度1型血管性血友病患者的首选药物，这些患者可通过它达到足够的血管性血友病因子（vWF）活性止血水平。由于存在输血相关病毒感染风险，随机供者的冷沉淀不再是最佳选择。在特殊情况下，来自非常小的供者库的冷沉淀，特别是在去氨加压素刺激后获得的冷沉淀，仍然是一种有吸引力的替代方案，因为这些供者可以进行密集筛查。这种治疗方法的成本可能也比商业浓缩物略低。目前，含有完整vWF多聚体的病毒减毒商业浓缩物是治疗中度至重度血管性血友病以及无法用去氨加压素充分治疗或去氨加压素禁忌的血管性血友病变异型患者以达到高水平vWF的首选治疗方法。人们希望专门设计用于治疗血管性血友病的vWF浓缩物将被证明是安全有效的。需要建立浓缩物中vWF的标准化检测方法。虽然尚未生产出最佳治疗产品，但最近开发的几种产品的结构更接近完整的正常血浆vWF，看起来很有前景。
