Management of Sickle Cell Disease-Related Pain in Children

描述(申请人提供)：镰状细胞病(SCD)是美国最常见的遗传性疾病之一。疼痛是儿童和成人SCD急性发病的最常见原因。疼痛发作是周期性的、自我限制的，但通常是令人痛苦的事件，如果治疗不当，可能会持续几个小时到几天，有时甚至几周。有关SCD相关疼痛的研究主要集中在成人，因此，国家心肺和血液研究所将急性疼痛的管理和预防确定为儿童研究的高度优先领域。对于患有SCD的儿童来说，大多数疼痛发作都是在家里进行管理的，但目前还没有研究在这种情况下对疼痛进行管理的对照研究。此外，越来越多的证据表明，疼痛经常在家中得不到足够的治疗。对于更严重的疼痛发作，SCD患者通常依赖急诊科(ED)的护理。尽管已公布的方案建议快速评估和治疗，但患有SCD的儿童在治疗疼痛方面经历了显著的延迟和变化。从PI与患有SCD儿童的父母的定性工作中，父母经常被在家中管理孩子的痛苦的前景所淹没。他们还报告说，他们的孩子在急诊室对SCD相关疼痛的治疗出现了延误。父母们相信，使用个性化的疼痛计划将提高照顾者预防和管理孩子SCD相关疼痛的能力。书面的、基于症状的管理计划已被证明在治疗慢性病方面有效，如哮喘和癌症相关疼痛。该项目的具体目标是：1)设计并进行一项全国性的儿童SCD或血液学项目主任和儿科急诊医学科主任的调查，以了解家庭和ED环境中是否以及如何使用疼痛方案或其他方法来管理SCD相关的儿童疼痛；2)召集专家小组，利用改进的Delphi方法制定预防和管理家庭和ED环境中儿童SCD相关急性疼痛的质量指标，并完善用于SCD儿童的个性化书面疼痛计划；3)对5至12岁的SCD儿童进行试点随机对照试验，以评估在家庭和ED环境中使用个性化书面疼痛计划的可行性和有效性。除了通过拟议的研究计划获得定量研究技能外，候选人还将获得患有SCD和疼痛的儿童的管理知识；通过额外的课程作业接受关于医疗保健的文化方面、高级生物统计学和研究设计的指导；以及了解照顾患有慢性病的儿童的系统方法。从这个项目中获得的信息和经验将被用来设计一项多中心随机对照试验，以检验个性化疼痛计划对SCD儿童的医疗保健利用、身体功能和生活质量的影响。

DESCRIPTION (provided by applicant): Sickle cell disease (SCD) is one of the most common genetic disorders in the US. Pain is the most common cause of acute morbidity in children and adults with SCD. Pain episodes are periodic, self-limited but often excruciating events that can last hours to days, sometimes weeks if inadequately treated. Studies on SCD-related pain have primarily focused on adults, therefore, the National Heart, Lung and Blood Institute identified acute pain management and prevention as a high-priority area for research in children. Most pain episodes are managed at home for children with SCD, yet there are no controlled studies examining the management of pain in this setting. In addition, there is growing evidence that pain is frequently under-treated at home. For more severe pain episodes, patients with SCD often rely on care in the emergency department (ED). Despite published protocols recommending rapid assessment and treatment, children with SCD experience significant delays and variation in the treatment of their pain. From the PI's qualitative work with parents of children with SCD, parents are often overwhelmed by the prospect of managing their child's pain at home. They also report delays in the treatment of their child's SCD- related pain in the ED. Parents believe that the use of an individualized pain plan would improve the caregiver's ability to prevent and manage their child's SCD-related pain. Written, symptom-based, management plans have been proven effective in treating chronic diseases, such as asthma and cancer-related pain. The specific aims of this project are to: 1) Design and conduct a national survey of directors of pediatric SCD or hematology programs and the division chiefs of pediatric emergency medicine to understand if, and how, pain protocols or other methods are used in the home and ED settings for the management of SCD- related pain in children; 2) Convene an expert panel to develop quality indicators for the prevention and management of acute SCD-related pain in children in the home and ED settings using the Modified Delphi method, and to refine an individualized written pain plan for use in children with SCD; and 3) Perform a pilot randomized controlled trial of children with SCD aged 5 to 12 years to evaluate the feasibility and efficacy of using an individualized written pain plan in the home and ED settings. In addition to acquiring quantitative research skills through the proposed research plan, the candidate will gain knowledge in the management of children with SCD and pain; receive instruction on the cultural aspects of health care, advanced biostatistics, and study design through additional coursework; and learn about systems approaches to caring for children with chronic illness. Information and experiences gained from this project will be used to design a multicenter randomized controlled trial to examine the effect of an individualized pain plan on the health care utilization, physical function, and quality of life of children with SCD.