Giangrande P, Seitz R, Behr-Gross M E, Berger K, Hilger A, Klein H, Schramm W, Mannucci P M
Oxford Haemophilia and Thrombosis Centre, Oxford, UK; European Haemophilia Consortium, Brussels, Belgium.
Haemophilia. 2014 May;20(3):322-5. doi: 10.1111/hae.12440.
This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the Kreuth III meeting in April 2013. To optimize the organization of haemophilia care nationally, it is recommended that a formal body be established in each country to include the relevant clinicians, national haemophilia patient organization, health ministry, paying authority and (if appropriate) regulatory authorities. The minimum factor VIII consumption level in a country should be 3 I.U. per capita. Decisions on whether to adopt a new product should not be based solely on cost. Prophylaxis for children with severe haemophilia is already recognized as the optimum therapy. Ongoing prophylaxis for individual adults should also be provided when required based on clinical decision making by the clinician in consultation with the patient. Children with inhibitors who have failed, or who are not suitable for, immune tolerance therapy should be offered prophylaxis with bypassing agents. Single factor concentrates should be used as therapy wherever possible in patients with rare bleeding disorders. Orphan drug designation for a factor concentrate should not be used to hinder the development, licencing and marketing of other products for the same condition which have demonstrably different protein modification or enhancement.
本报告总结了2013年4月在克吕特三世会议期间，36个欧洲国家的专家讨论得出的有关血友病治疗的建议。为了在全国范围内优化血友病护理的组织安排，建议各国成立一个正式机构，成员包括相关临床医生、国家血友病患者组织、卫生部、付费机构以及（如有必要）监管机构。一个国家的最低凝血因子VIII消耗量应为人均3国际单位。是否采用新产品的决策不应仅基于成本。对重度血友病患儿进行预防已被公认为最佳治疗方法。对于成年患者，也应根据临床医生与患者协商后做出的临床决策，在需要时提供持续预防治疗。对于免疫耐受治疗失败或不适合免疫耐受治疗的有抑制物的儿童，应使用旁路制剂进行预防。对于罕见出血性疾病患者，应尽可能使用单因子浓缩物进行治疗。不应将凝血因子浓缩物的孤儿药指定用于阻碍针对相同病症的其他产品的开发、许可和销售，这些产品具有明显不同蛋白质修饰或增强。
