School Readiness Intervention for Preschool Children with Sickle Cell Disease

项目摘要 安德鲁·海泽博士是圣裘德儿童研究医院心理学和生物行为科学系的助理成员。这项K23提案的总体目标是将Heitzer博士发展成为一名独立的研究者，专注于实施行为干预措施，以改善诊断为镰状细胞病（SCD）的患者的神经认知和学术成果。Heitzer博士组织了一个导师团队，他们拥有NIH资助的杰出记录，致力于推进他的职业生涯。导师在传播和实施科学，干预适应和发展，混合方法研究，并在SCD的行为临床试验的行为进行的专业知识。SCD是一种遗传性血红蛋白疾病，其特征是早期和进行性神经认知功能障碍，在美国影响10万人。普遍的神经认知困难显著导致数学、阅读和写作的学习成绩不佳。在制定循证干预措施以解决SCD认知缺陷的实质性功能后果方面存在重大差距。通过行为方法解决SCD神经认知缺陷的尝试一直难以坚持，限制了其有效性。消除依从性的障碍和增加可及性是干预成功的关键。对于SCD患者尤其如此，他们主要是来自服务不足社区的少数民族。这些家庭不太可能报告发育问题，在寻求帮助时经历更严重的耻辱，并且没有充分利用发育迟缓儿童的早期干预服务。生命早期的行为干预可以潜在地预防以后的神经发育和学术缺陷。具体而言，针对入学准备技能的干预措施，包括早期识字，算术和自我调节，在注意力，工作记忆和学习成绩方面产生持久的改善。虽然存在一些基于证据的入学准备干预措施，但他们没有考虑SCD患者特有的背景因素。具体目标是：1）调整行为干预，以改善学龄前SCD儿童的入学准备; 2）进行一项试点试验，与常规护理相比，调整入学准备干预，以评估可行性，可接受性和初步疗效。这将是第一项调整和测试适合SCD患者的干预方法的研究，有助于制定有针对性的干预措施，具有改善所有SCD儿童生活质量的巨大潜力。从试点试验中收集的数据将为申请R01资助的研究提供基础，以测试由St. Jude领导的正在进行的多中心纵向队列研究促进的干预措施的有效性。

PROJECT SUMMARY Andrew Heitzer, PhD, is an Assistant Member in the Psychology and Biobehavioral Sciences Department at St. Jude Children’s Research Hospital. The overall goal of this K23 proposal is the development of Dr. Heitzer into an independent investigator focused on implementing behavioral interventions to improve neurocognitive and academic outcomes in patients diagnosed with sickle cell disease (SCD). Dr. Heitzer has organized a mentorship team with distinguished records of NIH funding who are committed to advancing his career. Mentors have expertise in dissemination and implementation science, intervention adaptation and development, mixed-methods research, and conduct of behavioral clinical trials in SCD. SCD is a genetic disorder of hemoglobin characterized by early and progressive neurocognitive dysfunction affecting 100,000 individuals in the United States. Pervasive neurocognitive difficulties significantly contribute to poor academic performance in mathematics, reading, and writing. A critical gap exists in developing evidence-based interventions to address the substantial functional consequences of cognitive deficits in SCD. Attempts to address neurocognitive deficits in SCD through behavioral approaches have struggled with adherence, limiting their effectiveness. Addressing barriers to adherence and increasing accessibility is critical to intervention success. This is particularly true for patients with SCD, who are predominately racial minorities from underserved communities. These families are less likely to report developmental concerns, experience more significant stigma for seeking help, and underutilize early intervention services for children with developmental delays. Behavioral interventions early in life can potentially prevent later neurodevelopmental and academic deficits. Specifically, interventions that target school readiness skills, including early literacy, numeracy, and self-regulation, produce lasting improvements in attention, working memory, and academic performance. Although several evidence-based school readiness interventions exist, they have not considered the contextual factors unique to patients with SCD. The specific aims are: 1) Adapt a behavioral intervention to improve school readiness for preschoolers with SCD; 2) Conduct a pilot trial of the adapted school readiness intervention compared to routine care to assess feasibility, acceptability, and preliminary efficacy. This will be the first study to adapt and test an intervention approach fitted for patients with SCD, facilitating the development of targeted interventions with great potential for improving the quality of life for all children living with SCD. Data collected from the pilot trial will provide the foundation to apply for an R01-funded study to test the intervention’s efficacy facilitated by an ongoing multi-site longitudinal cohort study led by St. Jude.