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

项目总结： 该项目对临床、卫生政策和卫生经济问题进行创新的综合分析。 应用前瞻性成本-效果分析与镰状细胞病(SCD)的治疗相关 病人队列。该项目是及时的，对个人、提供者和卫生系统具有重要意义，因为 同种异体造血细胞移植(AllHCT)治疗SCD领域的快速发展现在需要进一步 在SCD治疗方案多种多样的背景下，对这种治疗方案进行调查。 具体地说，支持性护理方面的重大改进提高了异体血细胞移植治疗SCD的安全性和有效性 来自匹配的兄弟姐妹捐赠者(MSD)，导致这种移植的可接受性显著增加。这 导致出现了将allHCT扩大到没有MSD的个人使用的努力 候补捐赠者。与此同时，卫生政策正在改变异基因血细胞移植和治疗的格局。 对于SCD。2016年，CMS将SCD的allHCT纳入了全国覆盖范围确定，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 任务是了解异地HCT对#年健康、生活质量、资金和服务利用的影响 与已有的传统疗法相比，短期和长期治疗都是如此。这份K23提案计划解决 根据以下内容，对全面成本效益分析(CEA)的理解存在差距 步骤：1)定义已发表文献的过渡概率，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预期收集的PROMIS指标 在对CEA所需的基于偏好的效用分数的星级研究中进行的调查获得了预期 重大案例和控制的调整成本4)创建和分析包含所有数据的马尔可夫模型 从步骤1-3确定每种治疗策略的增量成本-效果比。我们还将 从患者的角度确定财务困难和服务利用率，以了解深度 这些疗法对患者和家属的影响。这项K23研究的结果将为 每种治疗对患者的健康、生活质量、财务和卫生系统的影响都将有助于 通过预测最优治疗策略进行个体决策。这些发现最终将成为 工具的基础是对疾病严重性和预后风险进行分层，并创建科学的方法来 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.