Bohn R L, Avorn J, Glynn R J, Choodnovskiy I, Haschemeyer R, Aledort L M
Program for the Analysis of Clinical Strategies, Brigham and Women's Hospital, Boston, MA 02115, USA.
Thromb Haemost. 1998 May;79(5):932-7.
Since the introduction of exogenous factor VIII therapy, several studies have explored the clinical benefits of prophylactic use of factor VIII. Little research, though, has focused on the economic aspects of this regimen. We conducted a cost analysis using data from the Orthopedic Outcomes Study, a prospective, cross-national study of the clinical outcomes associated with different patterns of factor VIII utilization to examine the health care costs incurred and expenditures averted in patients receiving on-demand versus prophylactic use of factor VIII in hemophilia.
831 patients with severe hemophilia aged 1 to 31 years, from 19 centers around the world were included in the cost analysis. Patients were categorized into three groups according to the number of weeks during the study years in which they received prophylactic regimens of factor VIII. For each subject, we estimated the costs of hospitalization, surgery, days lost from school or work, and factor VIII utilization. Costs were then stratified by age and by joint score to assess confounding, and a multivariate model developed to determine the relationship between use of factor VIII prophylaxis and total costs, while controlling for potential confounders.
Patients who received factor VIII episodically incurred substantially greater disability-related costs (days lost from school or work, days hospitalized due to hemophilia, surgery) than patients who received factor VIII prophylactically for some or all of the study period. For all treatment regimens, most disability-related costs were accounted for by hospitalization for hemophilia-related conditions. The cost of factor VIII itself was substantial in all treatment categories but was highest among patients who received year-round prophylaxis, exceeding the savings resulting from reduced disability and other health care expenditures.
Reductions in non-factor health care costs and disability associated with prophylactic use of factor VIII in hemophilia were substantial and helped somewhat to offset the much higher costs of this regimen. For certain subgroups, frequent episodic treatment may be more expensive than full-time prophylaxis. However, because of the very high cost of year-round prophylactic use of factor VIII, total health care expenditures were highest among patients receiving this therapeutic regimen. However, because prophylaxis clearly offers important clinical benefits, this approach may be warranted on medical rather than economic grounds.
自从引入外源性凝血因子 VIII 治疗以来，多项研究探讨了预防性使用凝血因子 VIII 的临床益处。然而，很少有研究关注该治疗方案的经济方面。我们利用骨科结局研究的数据进行了成本分析，这是一项前瞻性、跨国研究，涉及不同凝血因子 VIII 使用模式相关的临床结局，以检查血友病患者按需使用与预防性使用凝血因子 VIII 所产生的医疗保健成本和避免的支出。
成本分析纳入了来自全球 19 个中心的 831 例 1 至 31 岁的重度血友病患者。根据研究年度内接受凝血因子 VIII 预防性治疗方案的周数，将患者分为三组。对于每个受试者，我们估计了住院、手术、缺课或误工天数以及凝血因子 VIII 使用的成本。然后按年龄和关节评分对成本进行分层以评估混杂因素，并建立多变量模型以确定凝血因子 VIII 预防性使用与总成本之间的关系，同时控制潜在的混杂因素。
在研究期间，按需接受凝血因子 VIII 治疗的患者与部分或全部时间接受预防性凝血因子 VIII 治疗的患者相比，因残疾产生的成本（缺课或误工天数、因血友病住院天数、手术）要高得多。对于所有治疗方案，大多数与残疾相关的成本是由血友病相关疾病的住院费用构成。凝血因子 VIII 本身的成本在所有治疗类别中都很高，但在全年接受预防性治疗的患者中最高，超过了因残疾减少和其他医疗保健支出节省的费用。
血友病患者预防性使用凝血因子 VIII 可大幅降低非凝血因子医疗保健成本和残疾程度，并在一定程度上有助于抵消该治疗方案高得多的成本。对于某些亚组，频繁的按需治疗可能比全职预防性治疗更昂贵。然而，由于全年预防性使用凝血因子 VIII 的成本非常高，接受这种治疗方案的患者的总医疗保健支出最高。然而，由于预防性治疗显然具有重要的临床益处，从医学而非经济角度来看，这种方法可能是合理的。
