Smit C, Rosendaal F R, Varekamp I, Bröcker-Vriends A, Van Dijck H, Suurmeijer T P, Briët E
Department of Haematology, University Hospital Leiden, The Netherlands.
BMJ. 1989 Jan 28;298(6668):235-8. doi: 10.1136/bmj.298.6668.235.
A study was carried out among haemophiliacs in The Netherlands to evaluate the effect of modern substitution treatment (replacing the missing clotting factors) on medical and social performance. Three questionnaires were sent between 1972 and 1985. The use of prophylactic treatment in the group of patients with severe and moderately severe haemophilia increased from 21% (n = 242) in 1972 to 36% (n = 559) in 1985. Home treatment programmes increased from 4% to 53%. Overall mortality was 2.1 times higher than in the general male population, which leads to a calculated life expectancy of 66 years compared with 74 years in the general male population. Severe joint impairment was prominent in the older age groups, reflecting insufficient treatment in the past. A sharp decrease in the use of inpatient and outpatient hospital facilities was observed as well as much less absence from school and work. It is concluded that the high costs of modern substitution treatment are fully justified.
在荷兰的血友病患者中开展了一项研究，以评估现代替代疗法（补充缺失的凝血因子）对医疗和社会行为的影响。在1972年至1985年间发放了三份调查问卷。重度和中度重度血友病患者组中预防性治疗的使用比例从1972年的21%（n = 242）增至1985年的36%（n = 559）。家庭治疗方案的比例从4%增至53%。总体死亡率比普通男性人群高2.1倍，由此计算出的预期寿命为66岁，而普通男性人群为74岁。重度关节损伤在老年组中很突出，这反映出过去治疗不足。住院和门诊医院设施的使用大幅减少，同时缺勤和旷工情况也少得多。得出的结论是，现代替代疗法的高成本完全合理。
