Patient-centered home-based hematopoietic stem cell transplantation

 描述（由申请人提供）：造血干细胞移植（HCT）有可能治愈癌症和其他危及生命的疾病。然而，患者面临感染和其他并发症的长期脆弱性以及与治疗相关的大量死亡风险。因此，HCT通常发生在医院，住院时间持续三个月或更长。然而，住院治疗并非没有问题，包括医院感染，破坏稳态，增加资源利用和成本。以患者为中心的家庭移植（PCHT）是一种新的替代方案，可以提高患者的安全性，临床疗效和HCT患者的价值。PCHT首先对每个患者的需求进行全面评估。这包括与患者、护理人员、社会工作者、营养师、护士和医生会面。根据个性化的护理计划，临床医生每天早上在家中访问患者，进行评估并进行实验室检查。实验室在医院进行分析，护士返回患者家中提供所需的医疗干预措施（例如抗生素，输血）。利用我们的电子健康记录的力量，临床医生笔记本电脑允许远程访问患者的所有健康信息，潜在的药物相互作用和其他重要细节。此外，还为患者提供iPad，以便每天与医生进行视频会议，报告并发症或问题。我们已经成功地在HCT患者中试用了这种方法，证明了其可行性。通过允许患者呆在家里，PCHT可以减少医院病原体的暴露，减少医院感染。PCHT还可以减少移植物抗宿主病（GVHD），其影响40-60%的HCT患者并且是发病率和死亡率的主要原因。GVHD是由炎症驱动的，炎症可能是由肠道细菌的变化引发的。这种“肠道微生物群”受到环境变化（例如住院）的影响;然而，如果患者留在正常环境（家中），他们可能会保持正常的肠道微生物群，减少炎症和GVHD。呆在家里也可以促进独立，并通过允许更多地获得营养，锻炼和社会支持来提高生活质量（QOL）。虽然可能需要更密集的工作人员，但这些优点联合收割机可能会降低总成本。 本提案描述了一项比较PCHT与标准治疗的随机化II期研究。第一个目的是比较临床结果，包括GVHD（主要终点），感染，生存，生活质量和症状。还将评价这些结局的潜在介导因素（例如营养、运动、社会支持、自我效能）。第二个目的是评估这种方法的安全性，评估治疗相关的死亡率和不良事件。第三个目的是对直接医疗成本、卫生资源利用和间接成本（例如，患者时间）。长期目标是通过预防GVHD和感染等并发症来提高患者的安全性和寿命，促进QOL，并在提高护理质量的同时降低成本。

 DESCRIPTION (provided by applicant): Hematopoietic stem cell transplantation (HCT) has the potential to cure people with cancer and other life- threatening diseases. However, patients risk prolonged vulnerability to infections and other complications as well as substantial treatment-related mortality. Consequently, HCT typically occurs in the hospital, with stays lasting three months or more. Yet hospitalization is not without problems, including nosocomial infections, disrupted homeostasis, and increased resource utilization and costs. Patient-centered home transplant (PCHT) is a novel alternative that may improve patient safety, clinical efficacy, and value for HCT patients. PCHT begins with a comprehensive assessment of each patient's needs. This includes meetings with patients, caregivers, social workers, nutritionists, nurses, and physicians. Following an individualized care plan, clinicians visit patients in their homes every morning to perform assessments and draw labs. Labs are analyzed at the hospital, and nurses return to patient homes to deliver needed medical interventions (e.g. antibiotics, blood transfusions). Leveraging the power of our electronic health record, clinician laptops allow remote access to all of a patient's health information, potential drug interactions, and other important details. In addition, patients are provided with iPads to videoconference daily with physicians to report complications or concerns. We have successfully piloted this approach with HCT patients, demonstrating feasibility. By allowing patients to stay at home, PCHT may reduce exposure to hospital pathogens, decreasing nosocomial infections. PCHT may also reduce graft-versus-host disease (GVHD), which affects 40-60% of HCT patients and is a leading cause of morbidity and mortality. GVHD is driven by inflammation, which may be triggered by changes in the bacteria living in the gut. This "gut microbiota" is affected by changes in the environment (e.g. hospitalization); however, if patients stay in their normal environment (home), they may preserve their normal gut microbiota, decreasing inflammation and GVHD. Staying at home also may promote independence and improve quality of life (QOL) by allowing greater access to nutrition, exercise, and social support. While more intensive staffing may be needed, these advantages may combine to lower overall costs. This proposal describes a randomized phase 2 study to compare PCHT vs. standard care. The first aim is to compare clinical outcomes, include GVHD (primary endpoint), infections, survival, quality of life, and symptoms. Potential mediators of these outcomes (e.g. nutrition, exercise, social support, self -efficacy) will also be evaluated. The second aim is to evaluate the safety of this approach, assessing treatment-related mortality and adverse events. The third aim is to conduct an economic evaluation of the direct medical costs, health resources utilization, and indirect costs (e.g., patient time) with PCHT vs. standard care. The long-term objectives are to improve patient safety and longevity by preventing complications such as GVHD and infections, promote QOL, and lower costs while improving quality of care.