Plug Iris, van der Bom Johanna G, Peters Marjolein, Mauser-Bunschoten Evelien P, de Goede-Bolder Arja, Heijnen Lily, Smit Cees, Zwart-van Rijkom Jeannette E F, Willemse José, Rosendaal Frits R
Leiden University Medical Center, Clinical Epidemiology and the Thrombosis and Hemostasis Research Center, Leiden, The Netherlands.
Blood. 2004 Dec 1;104(12):3494-500. doi: 10.1182/blood-2004-05-2008. Epub 2004 Aug 12.
Since the introduction of replacement therapy in the early 1960s by the infusion of plasma-derived factor VIII and IX preparations, important changes have occurred for hemophilia patients. We studied the medical and social developments over 30 years of hemophilia treatment. Since 1972, 5 cross-sectional national postal surveys among all hemophilia patients in the Netherlands were performed, the latest in 2001. The prestructured questionnaires included items on treatment, the presence of inhibitory antibodies against factor VIII or IX, the annual number of bleeding episodes, use of inpatient hospital care, and hepatitis C and HIV infections. Response rate in 2001 was 70%. Young patients (<16 years) with severe hemophilia showed the largest increase in use of prophylaxis, from 34% in 1972 to 86% in 2001. The occurrence of hemorrhages has gradually decreased. Hospital admissions decreased from 47% of all patients in 1972 to 18% in 2001. Our study shows that the treatment of patients with severe hemophilia in the Netherlands has focused on the use of prophylactic treatment, especially in children. This has resulted in a decrease in bleeding frequency and an improvement of the medical and social circumstances of patients.
自20世纪60年代初通过输注血浆源性凝血因子VIII和IX制剂引入替代疗法以来，血友病患者的情况发生了重大变化。我们研究了血友病治疗30多年来的医学和社会发展情况。自1972年以来，对荷兰所有血友病患者进行了5次全国性横断面邮政调查，最近一次是在2001年。预先设计好的问卷包括治疗、针对凝血因子VIII或IX的抑制性抗体的存在情况、每年出血发作次数、住院治疗的使用情况以及丙型肝炎和艾滋病毒感染等项目。2001年的回复率为70%。重度血友病的年轻患者（<16岁）预防性治疗的使用率增长最大，从1972年的34%增至2001年的86%。出血的发生率逐渐下降。住院率从1972年所有患者的47%降至2001年的18%。我们的研究表明，荷兰重度血友病患者的治疗重点是预防性治疗的使用，尤其是在儿童中。这导致出血频率降低，患者的医学和社会状况得到改善。
