Cost Effectiveness Analysis of Stem Cell Transplant in Older MDS Patients

老年MDS患者干细胞移植的成本效益分析

• 批准号: 9252538
• 负责人: SCOTT D. RAMSEY
• 金额: $ 43.25万
• 依托单位: FRED HUTCHINSON CANCER RESEARCH CENTER
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-04-15 至 2019-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9252538
• 关键词: Address; Allogenic; Benefits and Risks; Blood; Bone Marrow Diseases; Bone Marrow Transplantation; Budgets; Caregivers; Cells; Clinical; Clinical Practice Guideline; Clinical Research; Clinical Trials; Clinical Trials Network; Comorbidity; Complement; Confidence Intervals; Cost Analysis; Cost Effectiveness Analysis; Data; Data Analyses; Data Collection; Decitabine; Development; Disease Progression; Dose; Dysmyelopoietic Syndromes; Economic Burden; Economics; Engraftment; Enrollment; Evaluation; Expenditure; Health; Health Insurance; Healthcare; Hematopoietic; Hematopoietic Stem Cell Transplantation; Hospitalization; Incidence; Infusion procedures; Inpatients; Insurance; Insurance Carriers; Insurance Coverage; Knowledge; Life; Malignant - descriptor; Marrow; Measures; Medical; Medicare; Medicine; Outcome; Outcome Assessment; Outpatients; Parents; Participant; Patient Recruitments; Patients; Preparation; Procedures; Provider; Quality of life; Quality-Adjusted Life Years; Records; Regimen; Relative Risks; Reporting; Resources; Risk; Schedule; Societies; Stem cell transplant; Supportive care; Surveys; Testing; Time; Transplantation; Uncertainty; United States Centers for Medicare and Medicaid Services; Work; aged; alternative treatment; arm; beneficiary; comparative; conditioning; cost; cost effective; cost effectiveness; curative treatments; economic impact; experience; graft vs host disease; hematopoietic cell transplantation; high risk; improved; interest; models and simulation; older patient; prospective; public health relevance; shared decision making; treatment strategy

 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.

 描述（由申请人提供）：项目摘要/摘要随着降低强度调节（RIC）方案的引入，同种异体造血干细胞移植（alloHCT）现在成为许多老年骨髓增生异常综合征（MDS）患者的可行治疗选择。作为 MDS 唯一的治愈性治疗选择，必须权衡与该手术相关的成本和风险，并与获得的潜在生命年进行比较，并与其他替代方案进行比较作为低甲基化药物（[HMA] 阿扎胞苷和地西他滨）的治疗，一项前瞻性、比较生物学分配试验 (BMT CTN 1102) 目前正在研究来自相关和无关供体的 RIC alloHCT 与 HMA 或中级 2/高患者的最佳支持治疗。 50-75 岁的风险 MDS 临床研究将检验以下假设：alloHCT am 的三年总生存率高于 HMA 和最佳支持治疗。鉴于 alloHCT 是当今医学上最昂贵的手术之一，BMT CTN 1102 的研究结果有可能扩大老年 MDS 患者获得 alloHCT 的机会和医疗保险覆盖范围，这对医疗保健支付者、患者和患者都有深远的影响。社会对 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，为进行比较 alloHCT 与 HMA 和最佳支持治疗的成本效益分析 (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，以补充母试验中评估的临床终点。可以在他们的护理人员经历治疗的经济后果时对其进行调查，减少回忆偏差并提高有效性。这是一个探索这两种替代方案的成本效益的时间敏感的机会，以便有效地从参与者那里收集前瞻性数据。在 BMT CTN 1102 中，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对 alloHCT 与母体研究相比的潜在成本效益进行全面评估的研究。健康管理协会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患者调查相结合，以获取自付费用和非医疗支出的结果。该辅助 CEA 的数据可供资源有限的患者、婴儿和健康保险公司使用，以协助决定替代 MDS 治疗策略。