South Carolina Study for Adolescents and Young Adults with Rare Conditions

南卡罗来纳州针对患有罕见疾病的青少年和年轻人的研究

• 批准号: 8240803
• 负责人: SUZANNE W MCDERMOTT
• 金额: $ 38.99万
• 依托单位: UNIVERSITY OF SOUTH CAROLINA AT COLUMBIA
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-30 至 2014-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8240803
• 关键词: South; Carolina; Study; Adolescents; Young

DESCRIPTION (provided by applicant): The proposed South Carolina project will be a collaboration between, the University of South Carolina, and the South Carolina State government (Budget and Control Board- Office for Research and Statistics). This project is possible because South Carolina has the potential to answer important questions about the health and welfare of adolescents and young adults with rare diseases using de-identified (anonymous) data from numerous agencies. For the 15-24 year age group, using only two data sources, the Hospital Discharge Data Set (HDDS) and Medicaid for the period 2000-2009, we have already identified 83 adolescents and young adults with fragile X syndrome, 635 with spinal bifida and 224 with muscular dystrophy. The project will have two parts: cross-sectional (a single point in time) and longitudinal (looking at people over an extended time period) and the study group will be 15-24 year olds with spina bifida, muscular dystrophy and fragile X syndrome. We will use linked administrative datasets (such as Medicaid, State Department of Education, and Vocational Rehabilitation) to describe the well-being and service use of the study group. We will estimate the prevalence of these conditions, their health status, educational, and employment participation. We will estimate the age specific prevalence for each condition and describe the occurrence of other acute and chronic medical conditions, including diabetes mellitus, obesity, heart disease, respiratory disease and other conditions. We will also describe the incidence and prevalence of co-morbid and secondary conditions and the co-occurrence of intellectual disability, paralysis, epilepsy, attention deficit hyperactivity disorder, and other conditions. We will also describe the frequency and type of health care received by each group, including rates of hospitalization, emergency department visits, and outpatient services, and the cost-effectiveness of different approaches to care. In the second and third year of the grant we will interview 125 affected individuals and their families to describe the living situation, education, employment, and other markers of social participation by adolescents and young adults with the three conditions, using the questionnaires from the National Longitudinal Transition Study and the RAND 36. We will describe the transition that occurs as adolescents with each condition mature into young adulthood, in terms of health care and social services (including type and specialty of health care provider by specialty, physical and occupational therapy, speech/language therapy, counseling and other services). Finally, we plan to assess the ability of other states to conduct the linked administrative data analyses and make recommendations that will be generalizable.

描述（由申请人提供）：提议的南卡罗来纳项目将是南卡罗来纳大学和南卡罗来纳州政府（预算和控制委员会-研究和统计办公室）之间的合作。这个项目之所以成为可能，是因为南卡罗来纳州有可能利用来自众多机构的去识别（匿名）数据，回答有关患有罕见疾病的青少年和年轻人的健康和福利的重要问题。对于15-24岁年龄组，仅使用2000-2009年期间的医院出院数据集（HDDS）和医疗补助两个数据来源，我们已经确定了83名患有脆性X染色体综合征的青少年和年轻人，635名患有脊柱裂，224名患有肌肉萎缩症。该项目将分为两部分：横断面（单个时间点）和纵向（在较长时间内观察人们），研究组将是15-24岁的脊柱裂、肌肉萎缩症和脆性X综合征患者。我们将使用相关的管理数据集（如医疗补助、州教育部和职业康复）来描述研究组的福祉和服务使用情况。我们将估计这些疾病的流行程度、他们的健康状况、教育和就业参与情况。我们将估计每种疾病的特定年龄患病率，并描述其他急性和慢性疾病的发生情况，包括糖尿病、肥胖、心脏病、呼吸系统疾病和其他疾病。我们还将描述合并症和继发性疾病的发生率和流行程度，以及智力残疾、瘫痪、癫痫、注意缺陷多动障碍和其他疾病的合并症。我们还将描述每个群体接受医疗保健的频率和类型，包括住院率、急诊科访问量和门诊服务，以及不同护理方法的成本效益。在赠款的第二年和第三年，我们将采访125名受影响的个人及其家庭，使用国家纵向过渡研究和兰德36的问卷，描述青少年和年轻人在这三个条件下的生活状况、教育、就业和其他社会参与标志。我们将从卫生保健和社会服务（包括按专业划分的卫生保健提供者的类型和专业、物理和职业治疗、言语/语言治疗、咨询和其他服务）的角度，描述患有每种疾病的青少年进入青年期时所发生的转变。最后，我们计划评估其他州进行相关行政数据分析的能力，并提出可推广的建议。