Early Identification Of Developmental Delay Among Infants And Toddlers With Sickle Cell Disease

早期识别患有镰状细胞病的婴儿和幼儿发育迟缓

• 批准号: 10590311
• 负责人: Catherine Rose Hoyt
• 金额: $ 12.74万
• 依托单位: WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-01 至 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10590311
• 关键词: 3 year old; Academy; Address; Adult; Affect; African ancestry; Age; Age Months; American; American Society of Hematology; Assessment tool; Award; Black race; Caregivers; Child; Child Development; Child Rearing; Child Welfare; Child health care; Clinical Research; Cognition; Cognitive; Collaborations; Complex; Data; Development; Development Plans; Developmental Delay Disorders; Diagnosis; Doctor of Philosophy; Early Intervention; Early identification; Education; Educational Curriculum; Educational Intervention; Environment; Evaluation; Evidence based intervention; Family; Family member; Funding; Future; Goals; Grant; Guidelines; Health; Healthcare Systems; Home; Home environment; Home visitation; Human; Impaired cognition; Incidence; Infant; Infant Development; Infection; Inflammation; Inherited; Institutional Racism; Intervention; Interview; Laboratories; Life; Longevity; Measures; Medical Care Team; Mendelian disorder; Mental Health; Mentored Patient-Oriented Research Career Development Award; Mentors; Methods; Missouri; Names; National Heart, Lung, and Blood Institute; Neuronal Plasticity; Newborn Infant; Nursery Schools; Occupational Therapist; Outcome; Outcome Measure; Pain; Parents; Pediatric Hospitals; Pediatrics; Persons; Pilot Projects; Population; Poverty; Preparation; Prevention; Protocols documentation; Randomized, Controlled Trials; Readiness; Recommendation; Rehabilitation therapy; Research; School-Age Population; Schools; Scientist; Severities; Sickle Cell; Sickle Cell Anemia; Stroke; Testing; Therapeutic Intervention; Time; Toddler; Training; Unemployment; United States; Visit; Work; career; career development; caregiver education; caregiver interventions; cognitive development; cohort; comparison control; comparison group; cost effective intervention; early experience; feasibility testing; implementation science; implementation strategy; improved; improved outcome; infancy; innovation; multidisciplinary; patient population; peer; programs; prospective; recruit; research and development; scale up; screening; service intervention; skills; social culture; standardize measure; success; teacher; theories; therapeutically effective; therapy development; trial design

PROJECT SUMMARY/ABSTRACT This K23 application proposes a research and career development plan for Catherine Hoyt, PhD, OTD to establish herself as an independent rehabilitation scientist focused on the early identification and intervention for developmental deficits among infants and toddlers with sickle cell disease (SCD). SCD is the most common monogenic disorder in humans and is primarily inherited by who identify as Black or of African descent. Complications associated with SCD (e.g., infection, pain, stroke) are common in the first years of life. In our earlier work, we found that developmental deficits were present in > 50% of children with SCD before the age of 3 but are none had been diagnosed or referred to intervention services. Further, children whose caregivers participated in a home-based caregiver education program demonstrated improved test scores on standardized measures. Thus, when developmental deficits are overlooked, children miss a critical opportunity for intervention that could improve their developmental trajectory. The American Society of Hematology (ASH) recommends frequent developmental screening starting in the first years of life for all children with SCD. Yet few, if any, studies have described the incidence and severity of developmental deficit among children with SCD compared to controls. Consistent with the American Academy of Pediatrics (AAP) guidelines, this research will evaluate children with SCD at 9, 18, and 30 months using the best available developmental measure, the Bayley Scales of Infant Development-4 (Bayley), to determine the incidence of developmental deficit over the first 3 years of life compared to demographically match peers (n = 100, Aim 1). If developmental deficits are identified, scores will be shared with the child's healthcare team so they can be addressed. Based on theory and evidence, the proposed study will also test a multi-component Sickle Cell Collaboration for Child Development (SCCCD) intervention. The SCCCD combines skilled therapeutic intervention to address developmental deficits, the Parents as Teachers® curriculum and specific SCD education. Our innovative SCCCD intervention is adapted from a pilot study and will provide 12 home visits to caregivers and children with SCD over the course of 1 year (n = 25, Aim 2). Interviews with caregivers who participated, as well as those who declined, will identify contextual determinants (i.e., facilitator and barriers) to prepare for future testing and scaling up of the SCCCD intervention (Aim 3). The results from this K23 award will provide data to understand the onset of developmental deficit in this understudied population and identify the next steps to conduct a randomized control trial to test our SCCCD intervention in an R01 level grant submission. These mentored research aims, combined with a career development plan for advanced training in implementation science (Goal A), mixed methods (Goal B), prospective trial design (Goal C) and professional development (Goals D, E) will enable Dr. Hoyt to launch a career as an independent scientist.

项目总结/摘要 这个K23应用程序提出了凯瑟琳霍伊特，博士，OTD的研究和职业发展计划， 建立自己作为一个独立的康复科学家专注于早期识别和干预 用于镰状细胞病（SCD）婴儿和幼儿的发育缺陷。SCD是最常见的 人类单基因疾病，主要由黑人或非洲血统的人遗传。 与SCD相关的并发症（例如，感染、疼痛、中风）在生命的最初几年是常见的。在我们 早期的研究发现，50%以上的SCD儿童在10岁以前就存在发育缺陷， 3人，但没有人被诊断或被转介到干预服务。此外，儿童的照顾者 参加了一个以家庭为基础的照顾者教育计划，证明了提高考试成绩， 标准化措施。因此，当发育缺陷被忽视时，孩子们就会错过一个关键的机会， 以改善他们的发展轨迹。美国血液学会（ASH） 建议对所有患有SCD的儿童在生命的最初几年开始进行频繁的发育筛查。然而 很少有研究（如果有的话）描述了儿童发育缺陷的发生率和严重程度， SCD与对照组相比。根据美国儿科学会（AAP）的指导方针， 研究将使用最好的发展方法评估9个月、18个月和30个月的SCD儿童。 Bayley婴儿发育量表-4（Bayley），以确定发育不良的发生率 与人口统计学上匹配的同龄人相比，在生命的前3年内存在缺陷（n = 100，目标1）。如果 发育缺陷被确定，分数将与儿童的医疗保健团队共享，以便他们可以 处理。基于理论和证据，拟议的研究还将测试多组分镰状细胞 儿童发展合作（SCCCD）。SCCCD结合了熟练的治疗 干预，以解决发展缺陷，家长作为教师®课程和具体的SCD 教育我们创新的SCCCD干预措施是根据试点研究改编的，将提供12次家访， 在1年的过程中，照顾者和患有SCD的儿童（n = 25，目标2）。与照顾者的访谈， 参与者，以及那些拒绝的人，将识别上下文决定因素（即，促进者和障碍） 为今后测试和扩大《荒漠化公约》的干预措施（目标3）做好准备。K23奖项的结果 将提供数据，以了解这一未充分研究的人群中发育缺陷的发生，并确定 接下来的步骤是进行随机对照试验，以测试我们在R 01级补助金中的SCCCD干预措施 成绩.这些指导的研究目标，结合职业发展计划，为高级培训 在实施科学（目标A），混合方法（目标B），前瞻性试验设计（目标C）和 专业发展（目标D，E）将使霍伊特博士作为一名独立的科学家开始职业生涯。