Purusothaman Vaishnavi, Ryther Robin C C, Bertrand Mary, Harker Lisa A, Jeffe Donna B, Wallendorf Michael, Smyth Matthew D, Limbrick David D
Division of Biology and Biomedical Sciences, Washington University in St. Louis Graduate School of Arts and Sciences, St. Louis, MO, USA.
Department of Neurology, Saint Louis Children's Hospital, Washington University in St. Louis School of Medicine, St. Louis, MO, USA.
Epilepsy Behav. 2014 Aug;37:26-31. doi: 10.1016/j.yebeh.2014.04.025. Epub 2014 Jun 23.
Up to 14% of children with epilepsy continue to experience seizures despite having appropriate medical therapy and develop medically refractory epilepsy (MRE). Assessing clinical outcomes and therapeutic efficacy in children with MRE undergoing palliative epilepsy surgery has been challenging because of the lack of a quantitative instrument capable of estimating the clinical status of these patients. The ideal instrument would at once consider seizure control, neurodevelopment, caregiver burden, and quality of life. The purpose of this study was to develop and pilot the Pediatric Refractory Epilepsy Questionnaire (PREQ), a quantitative instrument to assess the severity and individual burden of epilepsy in children with MRE undergoing palliative epilepsy treatments.
The caregivers of 25 patients with MRE completed the PREQ and the Quality of Life in Childhood Epilepsy (QOLCE) measure and participated in a semistructured interview. Medical records of the patients were reviewed, an Early Childhood Epilepsy Severity Scale (E-CHESS) score was calculated, and a Global Assessment of Severity of Epilepsy (GASE) score was obtained for each patient.
The initial PREQ was modified based on the analysis of responses, association with previously validated scales, comments from caregivers, and expertise of the PREQ panelists. Pediatric Refractory Epilepsy Questionnaire subscale scores were calculated based on clinical paradigm and compared with independent measures of seizure severity and quality of life. Significant correlations were observed between the seizure severity subscale and the GASE score (r=0.55) and between the mood subscale and the well-being score (r=0.61) on the QOLCE. Significant correlations were also observed between the caregiver rating of seizure severity and the GASE score (r=0.53), the social activity score (r=0.57), and the behavior score (r=0.43) on the QOLCE. Correlations between the caregiver rating of quality of life and the quality of life score (r=0.58) and the number of AEDs used (r=0.45) were also significant.
This pilot study is an initial, critical step in the development of the PREQ. The significant correlations between the PREQ subscales and the external epilepsy severity and quality of life measures lend preliminary support to our hypothesis that the PREQ is assessing the severity of epilepsy along with other important domains, such as mood, neurodevelopment, and quality of life. A larger prospective study of this modified PREQ is currently underway to further develop the PREQ.
高达14%的癫痫患儿尽管接受了适当的药物治疗仍继续发作，并发展为药物难治性癫痫（MRE）。评估接受姑息性癫痫手术的MRE患儿的临床结局和治疗效果一直具有挑战性，因为缺乏一种能够评估这些患者临床状况的定量工具。理想的工具应同时考虑癫痫发作控制、神经发育、照顾者负担和生活质量。本研究的目的是开发并试行儿童难治性癫痫问卷（PREQ），这是一种定量工具，用于评估接受姑息性癫痫治疗的MRE患儿癫痫的严重程度和个体负担。
25例MRE患儿的照顾者完成了PREQ和儿童癫痫生活质量（QOLCE）测评，并参与了半结构化访谈。查阅了患者的病历，计算了幼儿癫痫严重程度量表（E-CHESS）得分，并为每位患者获得了癫痫严重程度综合评估（GASE）得分。
根据对回答的分析、与先前验证量表的关联、照顾者的评论以及PREQ小组成员的专业知识，对初始PREQ进行了修改。基于临床范式计算了儿童难治性癫痫问卷分量表得分，并与癫痫严重程度和生活质量的独立测量指标进行了比较。在癫痫严重程度分量表与GASE得分之间观察到显著相关性（r = 0.55），在情绪分量表与QOLCE上的幸福感得分之间观察到显著相关性（r = 0.61）。在照顾者对癫痫严重程度的评分与GASE得分（r = 0.53）、社交活动得分（r = 0.57）以及QOLCE上的行为得分（r = 0.43）之间也观察到显著相关性。照顾者对生活质量的评分与生活质量得分（r = 0.58）以及使用的抗癫痫药物数量（r = 0.45）之间的相关性也很显著。
这项初步研究是PREQ开发过程中的关键第一步。PREQ分量表与外部癫痫严重程度和生活质量测量指标之间的显著相关性为我们的假设提供了初步支持，即PREQ正在评估癫痫的严重程度以及其他重要领域，如情绪、神经发育和生活质量。目前正在对这种修改后的PREQ进行更大规模的前瞻性研究，以进一步完善PREQ。
