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.

项目概要/摘要 此 K23 申请为 Catherine Hoyt 博士、OTD 提出了一项研究和职业发展计划，以 使自己成为一名独立的康复科学家，专注于早期识别和干预 治疗患有镰状细胞病（SCD）的婴儿和幼儿的发育缺陷。 SCD是最常见的 人类单基因疾病，主要由黑人或非洲人后裔遗传。 与 SCD 相关的并发症（例如感染、疼痛、中风）在生命的最初几年很常见。在我们的 在早期的研究中，我们发现超过 50% 的 SCD 儿童在 20 岁之前就存在发育缺陷 3 例，但无人被诊断或转介至干预服务机构。此外，儿童的照顾者 参加家庭护理人员教育计划后，测试成绩有所提高 标准化措施。因此，当忽视发育缺陷时，儿童就会错过一个重要的机会 寻求可以改善其发展轨迹的干预措施。美国血液学会 (ASH) 建议从出生后第一年开始对所有患有 SCD 的儿童进行频繁的发育筛查。然而 很少（如果有的话）研究描述了患有以下疾病的儿童发育缺陷的发生率和严重程度： SCD 与对照相比。与美国儿科学会 (AAP) 的指导方针一致， 研究将使用现有的最佳发育模型来评估 9、18 和 30 个月时患有 SCD 的儿童 婴儿发育贝利量表 4（Bayley），以确定发育障碍的发生率 与人口统计匹配的同龄人相比，生命前 3 年的赤字（n = 100，目标 1）。如果 确定发育缺陷后，分数将与孩子的医疗团队分享，以便他们能够 已解决。基于理论和证据，拟议的研究还将测试多成分镰状细胞 儿童发展合作 (SCCCD) 干预。 SCCCD 结合了熟练的治疗 解决发育缺陷的干预措施、Parents as Teachers® 课程和特定的 SCD 教育。我们创新的 SCCCD 干预措施改编自一项试点研究，将为患者提供 12 次家访 一年内患有 SCD 的护理人员和儿童（n = 25，目标 2）。采访护理人员 参与的人和拒绝参与的人将确定背景决定因素（即促进因素和障碍） 为未来测试和扩大 SCCCD 干预措施（目标 3）做好准备。本次 K23 奖项的结果 将提供数据来了解这一被研究人群中发育缺陷的发生情况并确定 接下来的步骤是进行随机对照试验，以测试我们在 R01 级别拨款中的 SCCCD 干预措施 提交。这些指导性研究目标与高级培训的职业发展计划相结合 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）将使霍伊特博士能够作为一名独立科学家开启职业生涯。