Long-term health-related quality of life and cost of allogeneic hematopoietic cell transplantation for children with sickle cell disease

项目概要： 该项目对临床、卫生政策和卫生经济问题进行了创新性的综合分析 相关的镰状细胞病（SCD）的治愈性治疗，使用前瞻性的成本效益分析 患者队列。该项目是及时的，对个人，提供者和卫生系统具有重要意义， 异基因造血细胞移植（alloHCT）治疗SCD的快速进展现在需要进一步研究。 在SCD治疗选择的不同背景下研究这种治疗选择。 特别是，支持性治疗的重大改进提高了alloHCT治疗SCD的安全性和有效性 来自匹配的同胞供体（MSD），导致这种移植的可接受性显著增加。这 已经导致出现了将alloHCT的可用性扩大到没有MSD的个体的努力， 替代捐赠者与此同时，卫生政策正在改变alloHCT和治愈性疗法的前景 关于SCD 2016年，CMS将SCD的alloHCT纳入国家覆盖范围确定，并有持续的证据 发展，包括侧重于病例对照研究和生活质量评估。因此，我们有一个明确的 了解alloHCT对健康、生活质量、财务和服务利用的影响， 短期和长期相比，已建立的传统疗法。K23计划解决 根据以下内容，通过全面的成本效益分析（CEA）来消除这种理解上的差距 步骤：1）从已发表的文献中定义转移概率，以作为基准或参考案例， 我们的前瞻性分析2）使用一种新的方法来翻译前瞻性收集的PROMIS措施 在STELLaR研究中调查了CEA所需的基于偏好的效用评分3）获得前瞻性 STELLaR病例和对照的调整成本4）创建并分析包含所有数据的马尔可夫模型 从步骤1-3中确定每种治疗策略的增量成本-效果比。我们还将 从病人的角度确定经济困难和服务利用率，以了解 这些疗法对患者和家庭的影响。这项K23研究的结果将为 每种治疗对患者健康、生活质量、财务和卫生系统的影响， 通过预测最佳治疗策略进行个人决策。这些发现最终将作为 对疾病严重程度和预后风险进行分层的工具的基础，以及创建一种科学方法， 研究其他儿科疾病。

Project summary: This project performs an innovative, integrated analysis of clinical, health policy, and health economic questions relevant to curative treatments for sickle cell disease (SCD) using cost-effectiveness analysis of a prospective patient cohort. The project is timely and of great significance to individuals, providers, and health systems as the rapid advances in the field of allogeneic hematopoietic cell transplantation (alloHCT) for SCD now warrant further investigation of this treatment option in the context of a varied landscape of therapeutic options for SCD. Specifically, critical improvements in supportive care have improved the safety and efficacy of alloHCT for SCD from a matched sibling donor (MSD) resulting in a dramatic increase in the acceptability of such transplants. This has resulted in the emergence of efforts to expand the availability of alloHCT to individuals without a MSD using alternate donors. At the same time, health policy is changing the landscape for alloHCT and curative therapies for SCD. In 2016, CMS included alloHCT for SCD in the national coverage determination with continued evidence development including focus on case control studies and quality of life assessment. Therefore, we have a clear mandate to understand the implications of alloHCT on health, quality of life, finances and service utilization in the short and long term compared to established conventional therapies. This K23 proposal plans to address this gap in understanding with a comprehensive cost-effectiveness analysis (CEA) according to the following steps: 1) define transition probabilities from published literature to serve as benchmarks or reference cases for our prospective analysis 2) use a novel methodology to translate prospectively collected PROMIS measures surveyed in the STELLaR study into preference based utility scores necessary for CEA 3) obtain prospective adjusted costs for STELLaR cases and controls 4) create and analyze a Markov model that incorporates all data from Steps 1-3 to determine the incremental cost-effectiveness ratio for each treatment strategy. We will also determine financial hardship and service utilization from a patient perspective in order to understand the depth of impact of these therapies on patients and families. The results of this K23 study will provide context for the affects of each treatment on patients' health, quality of life, finances, and the health system in a way that will aid individual decision-making by predicting the optimal treatment strategy. These findings will ultimately serve as the basis for tools to stratify disease severity and prognostic risk as well as create a scientific methodology to study other pediatric diseases.