Hood Anna M, Kölbel Melanie, Stotesbury Hanne, Kawadler Jamie, Slee April, Inusa Baba, Pelidis Maria, Howard Jo, Chakravorty Subarna, Height Sue, Awogbade Moji, Kirkham Fenella J, Liossi Christina
Developmental Neurosciences Unit and Biomedical Research Centre, University College London Great Ormond Street Institute of Child Health, London, United Kingdom.
Department of Primary Care and Population Health, University College London, London, United Kingdom.
Front Psychol. 2021 Sep 14;12:681137. doi: 10.3389/fpsyg.2021.681137. eCollection 2021.
Sickle cell disease (SCD) refers to a group of inherited blood disorders with considerable morbidity that causes severe pain, reduces life expectancy, and requires significant self-management. Acute painful episodes are the hallmark of SCD, but persistent daily pain is also highly prevalent in this population. Characterising the impact and experience of SCD-related morbidity (i.e., sleep disruption, frequent emergency department visits, cognitive dysfunction) on health-related quality of life (HRQOL) requires multiple assessment methods to best capture the underlying mechanisms. To gain a greater understanding of the effect of common symptom categories on HRQOL and to determine potential pain coping targets, the present study investigated whether demographic, socioeconomic, sleepiness, pain burden, frequency of emergency department (ED) visits, and cognition predicted HRQOL in a paediatric sample of patients with SCD. Our study was a secondary analysis of baseline assessment data of children with SCD aged 8-15 years ( = 30) in the Prevention of Morbidity in Sickle Cell Anaemia Phase 2b (POMSb2) randomised controlled clinical trial of auto-adjusting continuous positive airways pressure. Patients completed cognitive testing (IQ, Processing Speed Index, Delis-Kaplan Executive Function Scale (DKEFS) Tower, Conner's Continuous Performance Test), sleepiness (Epworth Sleepiness Scale), and HRQOL (PedsQL Sickle Cell Module) at baseline. Patients reported pain burden (Sickle Cell Pain Burden Inventory-Youth) each month over 8 visits. Caregivers provided demographic information and reported their child's executive function (Behavioural Rating Inventory of Executive Function) at baseline. Data from our analysis demonstrated that demographic factors (i.e., age, gender, level of neighbourhood deprivation) and treatment variables (i.e., hydroxyurea use) did not independently predict HRQOL, and laboratory values (i.e., haemoglobin, haematocrit, mean oxygen saturation) were not significantly correlated with HRQOL ( > 0.05). However, sleepiness, pain burden, ED visits, and executive dysfunction independently predicted HRQOL ( = 0.66) with large effects (η = 0.16 to 0.32). These findings identify specific, measurable symptom categories that may serve as targets to improve HRQOL that are responsive to change. This knowledge will be useful for multimodal interventions for paediatric patients with SCD that include sleep management, pain coping strategies, and executive function training.
镰状细胞病（SCD）是指一组遗传性血液疾病，其发病率相当高，会导致严重疼痛、缩短预期寿命，并且需要大量的自我管理。急性疼痛发作是SCD的标志，但持续性日常疼痛在该人群中也非常普遍。要描述SCD相关发病率（即睡眠中断、频繁急诊就诊、认知功能障碍）对健康相关生活质量（HRQOL）的影响和体验，需要多种评估方法来最好地捕捉潜在机制。为了更深入了解常见症状类别对HRQOL的影响并确定潜在的疼痛应对目标，本研究调查了在一个患有SCD的儿科患者样本中，人口统计学、社会经济状况、嗜睡程度、疼痛负担、急诊就诊频率和认知功能是否能预测HRQOL。我们的研究是对镰状细胞贫血症发病预防2b期（POMSb2）自动调节持续气道正压通气随机对照临床试验中8至15岁（n = 30）的SCD儿童基线评估数据的二次分析。患者在基线时完成了认知测试（智商、处理速度指数、德利斯科-卡普兰执行功能量表（DKEFS）塔楼测试、康纳持续性操作测试）、嗜睡程度评估（爱泼华嗜睡量表）和HRQOL评估（儿童生活质量量表镰状细胞模块）。患者在8次就诊期间每月报告疼痛负担（镰状细胞疼痛负担量表-青少年版）。照顾者在基线时提供人口统计学信息并报告其孩子的执行功能（执行功能行为评定量表）。我们分析的数据表明，人口统计学因素（即年龄、性别、邻里贫困程度）和治疗变量（即羟基脲的使用）并不能独立预测HRQOL，实验室指标（即血红蛋白、血细胞比容、平均氧饱和度）与HRQOL无显著相关性（p > 0.05）。然而，嗜睡程度、疼痛负担、急诊就诊次数和执行功能障碍能独立预测HRQOL（R² = 0.66），且影响较大（η² = 0.16至0.32）。这些发现确定了特定的、可测量的症状类别，这些类别可作为改善对变化有反应的HRQOL的目标。这一知识将有助于针对患有SCD的儿科患者的多模式干预措施，包括睡眠管理、疼痛应对策略和执行功能训练。
