Fischer K, Van Den Berg M
Department of Paediatrics, University Medical Center Utrecht, The Netherlands.
Haemophilia. 2003 Jul;9(4):376-81. doi: 10.1046/j.1365-2516.2003.00764.x.
Patients with severe haemophilia are treated either in case of bleeds only (on demand), or with regular infusions of clotting factor to prevent bleeds (prophylaxis). The introduction of prophylaxis has been hampered by issues of cost and viral safety. In order to compare results and treatment cost of different treatment strategies in adults, three cohorts of patients with severe haemophilia (born 1970-1980) were compared. 106 French patients were treated on demand, 49 Dutch patients were treated with intermediate dose prophylaxis, and 24 Swedish patients were treated with high dose prophylaxis. The annual number of joint bleeds, and the radiological Pettersson score were used to compare outcome, annual clotting factor consumption was used to compare costs. Prophylaxis reduced bleeds and arthropathy: patients treated on demand had a median of 11.5 joint bleeds/year and a median Pettersson score of 16 points, for intermediate dose prophylaxis median bleeds were 2.8 and Pettersson score was 7 points, and for high dose prophylaxis median bleeds were 0.5 joint bleeds and Pettersson score was 4 points. All differences were statistically significant, except the Pettersson scores in both prophylactic regimens. Treatment cost was only increased for high dose prophylaxis: mean clotting factor consumption was 1612 IU kg-1 yr-1 for on demand treatment, 1488 IU kg-1 yr-1 for intermediate dose prophylaxis, and 4012 IU kg-1 yr-1 for high dose prophylaxis. In young adults, the cost of intermediate dose prophylaxis is similar, but outcome is better than for on demand treatment. The cost of high dose prophylaxis is twofold higher, further improving outcome only slightly.
重度血友病患者仅在出血时（按需治疗）接受治疗，或定期输注凝血因子以预防出血（预防性治疗）。预防性治疗的推广受到成本和病毒安全性问题的阻碍。为了比较成人不同治疗策略的结果和治疗成本，对三组重度血友病患者（出生于1970 - 1980年）进行了比较。106名法国患者接受按需治疗，49名荷兰患者接受中等剂量预防性治疗，24名瑞典患者接受高剂量预防性治疗。用每年关节出血次数和放射学Pettersson评分来比较结果，用每年凝血因子消耗量来比较成本。预防性治疗减少了出血和关节病：按需治疗的患者每年关节出血中位数为11.5次，Pettersson评分中位数为16分；中等剂量预防性治疗的患者出血中位数为2.8次，Pettersson评分为7分；高剂量预防性治疗的患者出血中位数为0.5次，Pettersson评分为4分。除两种预防性治疗方案的Pettersson评分外，所有差异均具有统计学意义。治疗成本仅在高剂量预防性治疗时增加：按需治疗的平均凝血因子消耗量为1612 IU·kg⁻¹·年⁻¹，中等剂量预防性治疗为1488 IU·kg⁻¹·年⁻¹，高剂量预防性治疗为4012 IU·kg⁻¹·年⁻¹。在年轻成人中，中等剂量预防性治疗的成本相似，但结果优于按需治疗。高剂量预防性治疗的成本高出两倍，仅略微进一步改善了结果。
