Department of Psychology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Pediatr Blood Cancer. 2022 Mar;69(3):e29531. doi: 10.1002/pbc.29531. Epub 2021 Dec 31.
Children with sickle cell disease (SCD) experience neurodevelopmental delays; however, there is limited research with preschool-age children. This study examined neurocognitive risk and protective factors in preschoolers with SCD.
Sixty-two patients with SCD (60% HbSS/HbSβ -thalassemia; 40% HbSC/HbSβ -thalassemia) between the ages of 3 and 6 years (mean = 4.77 years) received a neuropsychological evaluation as routine systematic surveillance. Patients were not selected for disease severity, prior central nervous system findings, or existing cognitive concerns. Thirty-four patients (82% HbSS/HbSβ -thalassemia) were prescribed hydroxyurea (HU) at the time of their neuropsychological evaluation. On average, these patients had been prescribed HU at 2.15 (standard deviation = 1.45) years of age. The average dose was 28.8 mg/kg/day. Besides genotype, there were no group differences in medical or demographic factors based on HU treatment status.
Patients with HbSS/HbSβ -thalassemia scored below normative expectations on measures of intelligence, verbal comprehension, and school readiness (false discovery rate-adjusted p-value [p ] < .05). Age, sickle genotype, and HU treatment exposure were not associated with measured neurocognitive outcomes (p > .05). Greater social vulnerability at the community level was associated with poorer performance on measures of intellectual functioning, verbal comprehension, visuomotor control, and school readiness, as well as parent report of executive dysfunction (p < .05). Greater household socioeconomic status was positively associated with academic readiness.
Preschoolers with severe SCD (HbSS/HbSβ -thalassemia) perform below age expectations on measures of intelligence and academic readiness. Sociodemographic factors were stronger drivers of neurocognitive performance than disease severity or disease-modifying treatment. Neurodevelopmental interventions targeting the home and broader community environment are needed.
患有镰状细胞病 (SCD) 的儿童会出现神经发育迟缓；然而，针对学龄前儿童的研究有限。本研究旨在探讨 SCD 学龄前儿童的神经认知风险和保护因素。
62 名年龄在 3 至 6 岁（平均年龄=4.77 岁）的 SCD 患儿（60%为 HbSS/HbSβ-地中海贫血；40%为 HbSC/HbSβ-地中海贫血）接受了神经心理学评估作为常规系统监测。这些患儿并未根据疾病严重程度、中枢神经系统既往发现或现有认知问题进行选择。在进行神经心理学评估时，34 名患者（82%为 HbSS/HbSβ-地中海贫血）正在接受羟基脲 (HU) 治疗。这些患者平均在 2.15 岁（标准差=1.45）时开始服用 HU。平均剂量为 28.8mg/kg/天。除基因型外，根据 HU 治疗状况，两组在医疗或人口统计学因素方面无差异。
HbSS/HbSβ-地中海贫血患者在智力、语言理解和学业准备方面的得分低于正常值（经错误发现率校正后的 p 值 [p] <0.05）。年龄、镰状基因型和 HU 治疗暴露与测量的神经认知结果无关（p >0.05）。社区层面的社会脆弱性与智力、语言理解、视动控制和学业准备方面的表现以及父母报告的执行功能障碍呈负相关（p <0.05）。家庭社会经济地位较高与学业准备能力呈正相关。
严重 SCD（HbSS/HbSβ-地中海贫血）的学龄前儿童在智力和学业准备方面的表现低于年龄预期。社会人口统计学因素对神经认知表现的影响大于疾病严重程度或疾病修正治疗。需要针对家庭和更广泛的社区环境开展神经发育干预。
