Cost Effectiveness Analysis of Stem Cell Transplant in Older MDS Patients

 描述（由申请人提供）：项目总结/摘要随着降低强度预处理（RIC）方案的引入，异基因造血干细胞移植（alloHCT）现在是许多老年骨髓增生异常综合征（MDS）患者的可行治疗选择。虽然alloHCT是MDS的唯一治愈性治疗选择，但与该手术相关的成本和风险必须与获得的潜在生命年进行权衡，并与低甲基化药物（[HMA]阿扎他定和地西他滨）治疗等替代方案进行比较。一项前瞻性、比较性生物分配试验（BMT CTN 1102）目前正在研究来自相关和无关供体的RIC alloHCT与HMA或最佳支持治疗在50-75岁的中危-2/高危MDS患者中的对比。临床研究将测试的假设，即三年总生存率是较高的alloHCT上午比HMA和最佳支持性care arm. BMT CTN 1102的研究结果有可能扩大访问和医疗保险覆盖范围的alloHCT老年MDS患者。鉴于alloHCT是当今医学中最昂贵的程序之一，对医疗保健支付者，患者和社会都有深远的影响。alloHCT风险的不确定性， 特别是移植物抗宿主病和其他并发症，生活质量，以及与手术和潜在并发症治疗相关的高成本，为进行alloHCT与HMA和最佳支持治疗的成本效果分析（CEA）提供了令人信服的案例。与BMT CTN 1102一起进行辅助CEA提供了精确测量一系列生活质量和支出终点的机会，以补充母试验中评价的临床终点。通过前瞻性地将患者纳入CEA，可以在患者及其护理人员经历治疗的经济后果时对其进行调查，从而减少回忆偏差并提高有效性。这是一个时间紧迫的机会，可以探讨这两个备选方案的成本效益。为了有效收集BMT CTN 1102受试者的前瞻性数据，辅助CEA将与母研究合作招募患者并遵循类似的数据收集时间表。总之，拟议的研究将是第一个全面评估alloHCT与HMA相比的潜在成本效益以及治疗老年MDS患者的最佳支持护理的研究。为了从多个角度评估这两种策略的经济影响，保险索赔记录将与直接的患者调查相结合，以获取自付和非医疗支出。这一辅助CEA的结果可用于患者，临床医生和健康保险公司在有限的资源下工作，以帮助决定替代MDS治疗策略。

 DESCRIPTION (provided by applicant): PROJECT SUMMARY/ABSTRACT With the introduction of reduced intensity conditioning (RIC) regimens, allogeneic hematopoietic stem cell transplantation (alloHCT) is now a viable treatment option for many older myelodysplastic syndrome (MDS) patients. While alloHCT is the only curative treatment option for MDS, the costs and risks associated with the procedure have to be weighed against potential life-years gained and compared to alternatives such as treatment with hypomethylating agents ([HMA] azacitadine and decitabine). A prospective, comparative biologic assignment trial (BMT CTN 1102) is now studying RIC alloHCT from related and unrelated donors versus HMA or best supportive care among patients with Intermediate-2/High risk MDS aged 50-75 years. The clinical study will test the hypothesis that three-year overall survival is higher in the alloHCT am than in the HMA and best supportive care arm. The findings of BMT CTN 1102 have the potential to expand access to and Medicare coverage of alloHCT to older MDS patients. Given that alloHCT is one of the costliest procedures in medicine today, there are profound implications for health care payers, patients, and society. Uncertainty about the risks of alloHCT, specifically graft versus host disease and other complications, quality of life, and its high cost associated with the procedure and the treatment of potential complications provide a compelling case for conducting a cost- effectiveness analysis (CEA) comparing alloHCT to HMA and best supportive care. Conducting an ancillary CEA alongside BMT CTN 1102 provides the opportunity to precisely measure a range of quality of life and expenditure endpoints to complement the clinical endpoints evaluated in the parent trial. By prospectively enrolling patients into the CEA, patients and their caregivers can be surveyed while they are experiencing the economic consequences of treatment, reducing recall bias and enhancing validity. This is a time-sensitive opportunity to explore the cost-effectiveness of these two alternative options. In order to efficiently collect prospective data from participants in BMT CTN 1102, the ancillary CEA will work with the parent study to recruit patients and follow a similar data collection schedule. In summary, the proposed study will be the first to provide a comprehensive evaluation of the potential cost- effectiveness of alloHCT compared to HMA and best supportive care for the treatment of older MDS patients. To evaluate the economic Impacts of these two strategies from multiple perspectives, insurance claims records will be combined with direct patient surveys, to capture out-of-pocket and non-medical expenditures. The findings of this ancillary CEA may be used by patients, clinicians, and health insurers working with limited resources to assist in decisions about alternative MDS treatment strategies.