A Vision of Hope: Integration of Palliative Care in Chronic Pediatric Diseases

描述（由申请人提供）：在广泛的挑战领域（04）临床研究下，本提案直接响应以下具体挑战主题：04-NR-102* 加强姑息治疗和临终研究的方法本倡议将开发和测试干预措施，以提高对危及生命的疾病患者的护理质量。这项研究将为制定循证指南以规范姑息治疗和临终关怀提供基础。儿童、青少年和年轻人不应该患重病或死亡，但一旦他们患上重病或死亡，他们的生活和家庭将永远改变。除了患有慢性、危及生命的疾病的年轻人所面临的身体挑战之外，许多这些疾病的不可预测性和限制生命的性质给受影响的个人及其家庭带来了重大的情感、精神和社会困难，并给卫生保健团队带来了特殊的挑战。往往，支离破碎的医疗保健提供系统和缺乏支持系统增加了痛苦。这些挑战在至少目前没有治愈性治疗方法的疾病中尤为突出。没有治疗方法可能会导致失去希望。该提案旨在通过将姑息治疗的原则和做法纳入对护理他们的临床医生的培训，提高护理质量，并为受慢性、危及生命疾病影响的青少年、年轻人和家庭灌输新的希望愿景。该项目特别关注两种遗传疾病，杜氏肌营养不良症（DMD）和镰状细胞病（SCD）。虽然这些疾病之间存在显着差异，但两者都危及生命和限制生命，因此非常适合姑息治疗。改进的技术和医疗干预措施显着增加了受影响的个人的预期寿命，但这也带来了新的挑战，从儿科到成人护理临床医生和设置的患者过渡。我们项目的具体目标是：（1）了解患有这些慢性、危及生命的疾病的青少年和年轻人的生活经历和未满足的需求，以及照顾这些人群对跨学科临床医生团队的影响;（2）通过培训跨学科专业人员、患者和家庭，加强姑息治疗原则和实践的整合。我们将分三个阶段实现这些目标。首先，我们将根据对患者、家属和护理他们的临床医生的采访，开发和/或修改记录培训视频。第二，我们将调整现有的培训计划，将姑息治疗原则纳入这两个人群的护理。最后，我们将比较/评估两种不同的培训干预措施在改善临床医生的态度和行为，以及患者的经验，姑息治疗的几个组成部分的有效性。该项目可能会对减少综合姑息治疗的态度和系统障碍产生重大影响。我们的研究结果将为教育和实践指南的完善提供信息，这些指南将姑息治疗原则整合到DMD和SCD护理中，并将其应用于其他限制生命的慢性病，并导致长期改善为患有慢性病的年轻人提供优质医疗保健。该提案旨在通过将姑息治疗的原则和实践纳入对护理他们的临床医生的培训中，为受杜氏肌营养不良症（DMD）和镰状细胞病（SCD）这两种最常见的慢性遗传性危及生命的疾病影响的青少年、年轻人和家庭提高护理质量，并灌输新的希望愿景。

DESCRIPTION (provided by applicant): Under the broad challenge area (04) Clinical Research, this proposal is directly responsive to the following specific challenge topic: 04-NR-102* Methods to Enhance Palliative Care and End-of-Life Research This initiative will develop and test interventions to enhance the quality of care for persons with a life-threatening illness. This research will provide the foundation for the development of evidenced-based guidelines to standardize palliative and end-of-life care. Children, adolescents and young adults are not supposed to get seriously ill or die, but when they do, their lives and their families are forever changed. In addition to the physical challenges faced by young people with chronic, life-threatening diseases, the unpredictability and life- limiting nature of many of these diseases raise significant emotional, spiritual and social difficulties for affected individuals and their families, and create special challenges for the health care team. Too often, a fragmented health care delivery system and lack of support systems add to the distress. These challenges are particularly salient in the context of diseases that, at least currently, have no curative treatments. The absence of a cure can result in a loss of hope. This proposal is designed to improve the quality of care, and instill a new vision of hope, for adolescents, young adults and families affected by chronic, life-threatening diseases by integrating the principles and practices of palliative care into the training of the clinicians who care for them. This project is specifically focused on two genetic conditions, Duchenne Muscular Dystrophy (DMD) and Sickle Cell Disease (SCD). Although there are significant differences between these conditions, both are life-threatening and life-limiting and are therefore highly amenable to palliative care. Improved technologic and medical interventions have significantly increased the life expectancy of affected individuals but this has created new challenges with regard to transitioning patients from pediatric to adult care clinicians and settings. The specific aims of our project are to (1) understand the lived experiences and unmet needs of adolescents and young adults with these chronic, life-threatening diseases, and the impact of caring for these populations on interdisciplinary teams of clinicians; and (2) enhance the integration of palliative care principles and practices through the training of interdisciplinary professionals, patients and families. We will accomplish these aims in three phases. First, we will develop and/or modify documentary training videos based on interviews with patients, families and the clinicians who care for them. Second, we will adapt an existing training program that incorporates palliative care principles in the delivery of care for these two populations. Finally, we will compare/assess the efficacy of two different training interventions in improving clinician attitudes and behaviors, and patient experiences, with respect to several components of palliative care. This project is likely to have significant impact on reducing the attitudinal and systemic barriers to the integration of comprehensive palliative care. Our results will inform the refinement of educational and practice guidelines with regard to the integration of palliative care principles into DMD and SCD care as well as its application to other life-limiting chronic conditions, and result in long term improvements in the delivery of quality health care for young people with chronic disease. This proposal is designed to improve the quality of care, and instill a new vision of hope, for adolescents, young adults and families affected by Duchenne Muscular Dystrophy (DMD) and Sickle Cell Disease (SCD), two of the most common chronic, inherited, life- threatening diseases, by integrating the principles and practices of palliative care into the training of the clinicians who care for them.