Shrestha A, Eldar-Lissai A, Hou N, Lakdawalla D N, Batt K
Precision Health Economics, Los Angeles, CA, USA.
Biogen, Cambridge, MA, USA.
Haemophilia. 2017 Jul;23(4):e267-e275. doi: 10.1111/hae.13220. Epub 2017 Jun 2.
Prophylaxis treatment is recommended for haemophilia patients, but associated real-world economic costs and potential cost-savings associated with improved disease management are not fully known. This study aimed to assess haemophilia A-related resource use and cost by treatment type (prophylaxis versus non-prophylaxis) and any associated cost-savings.
Truven MarketScan Commercial claims data (2004-2012) were used to identify haemophilia A-related healthcare utilization, healthcare costs and patterns of prophylaxis and non-prophylaxis treatment among 6- to 64-year-old males. We estimated bleeding-related resource utilization and costs in three age groups (6-18, 19-44, 45-64) by treatment types and assessed the extent to which early initiation of prophylactic treatment can mitigate them. T-tests and ordinary least squares regressions were used to compare unadjusted and demographics-adjusted cost estimates.
Among children, overall haemophilia- and bleeding-related non-pharmacy costs were substantially lower for patients receiving prophylaxis (haemophilia-related: $15,864 vs. $53,408; P < 0.001; bleeding-related: $696 vs. $2013, respectively; P = 0.04). Among younger adults (19-44), haemophilia-related non-pharmacy costs were lower for patients receiving prophylaxis ($22,028 vs. $56,311, respectively; P = 0.001). Among children, these savings fully offset the incremental pharmacy cost due to prophylaxis. Among younger adults, the savings offset approximately 34% of the incremental pharmacy cost. No differences were found for older adults (45-64).
These results suggest that initiating prophylaxis earlier in life may reduce the healthcare costs of bleeding events and their long-term complications. Future studies should strive to collect more detailed information on disease severity and treatment protocols to improve estimates of disease burden.
推荐对血友病患者进行预防性治疗，但实际的经济成本以及疾病管理改善带来的潜在成本节约情况尚不完全清楚。本研究旨在按治疗类型（预防性治疗与非预防性治疗）评估甲型血友病相关的资源使用和成本以及任何相关的成本节约情况。
使用Truven MarketScan商业索赔数据（2004 - 2012年）来确定6至64岁男性中与甲型血友病相关的医疗保健利用情况、医疗成本以及预防性和非预防性治疗模式。我们按治疗类型估计了三个年龄组（6 - 18岁、19 - 44岁、45 - 64岁）中与出血相关的资源利用和成本，并评估了早期开始预防性治疗可减轻这些情况的程度。采用t检验和普通最小二乘法回归来比较未经调整和经人口统计学调整后的成本估计值。
在儿童中，接受预防性治疗的患者与血友病及出血相关的总体非药物成本显著更低（与血友病相关的成本：15,864美元对53,408美元；P < 0.001；与出血相关的成本：分别为696美元对2013美元；P = 0.04）。在年轻成年人（19 - 44岁）中，接受预防性治疗的患者与血友病相关的非药物成本更低（分别为22,028美元对56,311美元；P = 0.001）。在儿童中，这些成本节约完全抵消了预防性治疗带来的额外药物成本。在年轻成年人中，成本节约抵消了约34%的额外药物成本。在老年人（45 -
64岁）中未发现差异。
这些结果表明，在生命早期开始预防性治疗可能会降低出血事件及其长期并发症的医疗成本。未来的研究应努力收集关于疾病严重程度和治疗方案的更详细信息，以改进疾病负担的估计。
