BC Cancer, Department of Medical Oncology, Vancouver, Canada.
DRG Abacus, Manchester, United Kingdom.
Gynecol Oncol. 2019 Apr;153(1):87-91. doi: 10.1016/j.ygyno.2019.01.018. Epub 2019 Jan 28.
Survival but not cure rates have improved for epithelial ovarian cancer (EOC), demonstrating the need for effective prevention. Targeted prevention in BRCA carriers by risk reducing surgery (RRS) prevents 80% of cases but incurs additional up-front costs, compensated for by the potential for long term savings from treatment avoidance. Does prevention represent value for money? In the absence of long-term data from prospective trials, determining the cost effectiveness of a prevention strategy requires economic modelling.
A patient level simulation was developed comparing outcomes between two groups, using Canadian data. Group 1: no mutation testing with treatment if EOC developed. Group 2: cascade testing (index patient BRCA tested and the first and second-degree relatives tested if index patient or first-degree relative respectively were positive) with RRS in carriers. End points were Incremental Cost-Effectiveness Ratio (ICER) and budget impact.
2786 women with EOC (1 year incidence) had 766 first and 207 second-degree female relatives. BRCA mutations were present in 390 index cases, 366 first and 49 second-degree relatives. With 100% RRS uptake, 59 EOC were prevented and testing dominated no testing (more effective and less costly; ICER -$8919). The total cost saving over 50 years was $2,904,486 (cost saving of $9,660,381 in treatment costs versus increased cost from cascade testing/RRS of $6,755,895). At a threshold of $100,000 per QALY, prevention was cost effective in all modelled scenarios.
Targeted prevention in BRCA mutation carriers not only prevents EOC but is cost-effective compared to treating EOC if it develops.
上皮性卵巢癌(EOC)的生存率而非治愈率有所提高,这表明需要采取有效的预防措施。通过风险降低手术(RRS)对 BRCA 携带者进行靶向预防可预防 80%的病例,但会增加前期成本,而通过避免治疗可获得长期节省来弥补潜在成本。预防措施是否具有成本效益?在缺乏前瞻性试验的长期数据的情况下,确定预防策略的成本效益需要进行经济建模。
使用加拿大数据,通过患者水平模拟,比较了两组患者的结局。第 1 组:不进行突变检测,如果发生 EOC 则进行治疗。第 2 组:进行级联检测(对索引患者进行 BRCA 检测,如果索引患者或一级亲属为阳性,则对一级和二级亲属进行检测),携带者进行 RRS。终点为增量成本效益比(ICER)和预算影响。
2786 名患有 EOC(1 年发病率)的女性中,有 766 名一级和 207 名二级女性亲属。390 名索引病例、366 名一级亲属和 49 名二级亲属存在 BRCA 突变。如果 100%接受 RRS,可预防 59 例 EOC,且检测优于不检测(更有效且成本更低;ICER 为-8919 美元)。在 50 年内,总节省成本为 2904486 美元(治疗成本节省 9660381 美元,而由于级联检测/RRS 增加的成本为 6755895 美元)。在 10 万美元/QALY 的阈值下,预防在所有模型情景中均具有成本效益。
在 BRCA 突变携带者中进行靶向预防不仅可预防 EOC,而且与治疗 EOC 相比具有成本效益。