Fischer K, Astermark J, van der Bom J G, Ljung R, Berntorp E, Grobbee D E, van den Berg H M
Department of Paediatrics, University Medical Center Utrecht, The Netherlands.
Haemophilia. 2002 Nov;8(6):753-60. doi: 10.1046/j.1365-2516.2002.00694.x.
A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg-1 year-1. Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year-1 vs. 3.3 year-1) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up.
在瑞典和荷兰开展了一项多中心研究，比较了两种预防方案对1970年至1990年出生的128例重度血友病患者的影响。42例瑞典患者（高剂量预防组）与86例荷兰患者（中剂量预防组）进行了比较。根据Pettersson标准，在患者最后一次影像学评分时对其进行评估。在评估前的三年期间记录年度凝血因子消耗量和出血频率。高剂量组患者在评估时年龄更小（中位数分别为15.2岁和17.9岁），开始预防的时间更早（中位数分别为2岁和5岁），每年每千克体重使用的凝血因子量多出2.19倍。接受高剂量预防治疗的患者关节出血较少（中位数分别为每年0.3次和3.3次），根据Pettersson评分，无关节病的患者比例更高（分别为69%和32%），然而，年龄调整后的评分差异较小（中位数分别为0分和4分），目前无统计学意义。临床评分和生活质量相似。这些研究结果表明，与中剂量预防相比，高剂量预防在3年期间显著增加了治疗成本并减少了关节出血，但在随访17年后仅略微降低了关节病的发生率。
