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，本提案直接回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这一倡议将开发和测试干预措施，以提高对患有危及生命的疾病的人的护理质量。这项研究将为制定以证据为基础的指南以标准化姑息和临终关怀提供基础。儿童、青少年和年轻人不应该得重病或死亡，但当他们得了重病或死亡时，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面临的身体挑战外，其中许多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人带来了严重的情感、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，分散的医疗保健提供系统和缺乏支持系统增加了痛苦。这些挑战在疾病的背景下尤其突出，至少在目前，这些疾病没有根治方法。没有治愈方法可能会导致希望的丧失。这项建议旨在通过将姑息治疗的原则和做法纳入护理他们的临床医生的培训，为青少年、青壮年和受慢性、危及生命的疾病影响的家庭提高护理质量，并向他们灌输新的希望。这个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)。尽管这两种情况有很大的不同，但两者都是危及生命和限制生命的，因此非常适合姑息治疗。改进的技术和医疗干预措施大大延长了受影响个人的预期寿命，但这在将患者从儿科护理过渡到成人护理、临床医生和环境方面带来了新的挑战。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得到满足的需求，以及护理这些人群对临床医生跨学科团队的影响；以及(2)通过培训跨学科的专业人员、患者和家庭，加强姑息治疗原则和做法的整合。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/或修改纪实培训视频。其次，我们将调整现有的培训计划，在为这两个群体提供护理时纳入姑息治疗原则。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态度和行为以及患者体验方面的有效性，涉及姑息治疗的几个组成部分。这一项目可能会对减少综合姑息治疗的态度和体制障碍产生重大影响。我们的结果将有助于完善关于将姑息治疗原则纳入DMD和SCD护理以及将其应用于其他限制生命的慢性疾病的教育和实践指南，并导致长期改善为患有慢性病的年轻人提供高质量的卫生保健。这项建议旨在通过将姑息治疗的原则和做法融入护理他们的临床医生的培训中，为受Duchenne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、年轻人和家庭改善护理质量，并向他们灌输新的希望。

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.