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

项目摘要/摘要 此K23应用程序为凯瑟琳·霍伊特博士和OTD提出研究和职业发展计划，以 将自己确立为一名独立的康复科学家，专注于早期识别和干预 针对患有镰状细胞病(SCD)的婴幼儿的发育缺陷。SCD是最常见的 人类中的单基因疾病，主要由认同为黑人或非洲后裔的人遗传。 与SCD相关的并发症(如感染、疼痛、中风)在生命的头几年很常见。在我们的 早期的工作，我们发现50%的SCD儿童在这个年龄之前就存在发育缺陷 3个，但没有一个被诊断或转诊到干预服务机构。此外，照顾者的孩子们 参加了以家庭为基础的照顾者教育计划，表现出考试成绩的提高 标准化措施。因此，当发育缺陷被忽视时，儿童就会错失一个关键的机会。 进行干预，以改善他们的发展轨迹。美国血液病学会(ASH) 建议对所有患有SCD的儿童从出生后的第一年开始进行频繁的发育筛查。还没有 很少，如果有的话，研究描述了儿童发育缺陷的发生率和严重性 SCD与对照组比较。这与美国儿科学会(AAP)的指导方针一致 研究将在9个月、18个月和30个月时使用可用的最佳发育评估方法评估患有SCD的儿童 测量，贝利婴儿发育量表-4(贝利)，以确定发育不良的发生率 与人口统计上匹配的同龄人(n=100，目标1)相比，前3年的出生缺陷。如果 发育缺陷被识别出来，分数将与儿童的医疗团队分享，这样他们就可以 地址。在理论和证据的基础上，拟议的研究还将测试一种多组分的镰刀电池 合作促进儿童发展(SCCD)干预。SCCD结合了熟练的治疗 解决发展缺陷的干预措施、家长作为教师的课程和特定的SCD 教育。我们创新的SCCD干预措施改编自一项试点研究，将提供12次家访，以 1年以上的照顾者和患有SCD的儿童(n=25，目标2)。对照顾者的采访 参与的人以及拒绝参加的人将确定背景决定因素(即促进者和障碍)，以 为未来SCCD干预措施的测试和扩大做好准备(目标3)。K23大奖的评选结果 将提供数据，以了解在这一研究不足的人群中出现发育缺陷的原因，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水平拨款中的SCCD干预 呈件。这些受指导的研究目标，与高级培训的职业发展计划相结合 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 职业发展(目标D、E)将使霍伊特博士能够开始独立科学家的职业生涯。

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.