Azevedo Sara, Oliveira Maria Miguel, Nogueira Paulo, Lopes Ana Isabel
Gastroenterology Unit, Pediatrics Department, Santa Maria University Hospital-CHLN, Academic Medical Centre of Lisbon, Lisbon, Portugal.
Pediatrics University Clinic, Medical School, University of Lisbon, Lisbon, Portugal.
Front Pediatr. 2025 Jan 15;12:1473286. doi: 10.3389/fped.2024.1473286. eCollection 2024.
Inflammatory bowel disease (IBD) may adversely affect physical, psychological, and social well-being. Integrating patient-reported outcomes (PROs) into clinical practice is crucial for comprehensive disease management.
To evaluate the responsiveness and clinical utility of Patient-Reported Outcomes Measurement Information System (PROMIS) instruments, compared with standard clinical assessment tools in pediatric CD patients.
A longitudinal, prospective study with 31 pediatric Crohn's disease (CD) patients aged 8-17 years recruited from a Pediatric Gastroenterology Unit. Data were collected at baseline and every 6 months over 18 months. PROMIS pediatric measures assessed PROs. Disease activity was evaluated using the pediatric Crohn's disease activity index (PCDAI) and clinical markers. IMPACT-III was also applied. Linear mixed-effects models (LMMs) and bivariate analyses were used to assess changes over time.
PROMIS Global Health scores showed significant improvement over time, indicating enhanced overall health perceptions among patients. Notable reductions were observed in PROMIS Pain Interference and Fatigue scores, indicating better physical health. PROMIS depression scores generally decreased, suggesting improved mental health. PCDAI scores, hemoglobin, and platelet count significantly changed and correlated with PROMIS measures. Globally, the study demonstrated significant and clinically relevant changes in multiple PROMIS measures, confirming their responsiveness to changes in disease activity.
PROMIS instruments are clinically useful in managing pediatric CD, providing valuable insights into global health and quality of life. Integrating PROMIS measures into routine clinical practice may enhance disease management and treatment strategies for pediatric IBD patients.
炎症性肠病（IBD）可能对身体、心理和社会福祉产生不利影响。将患者报告的结局（PROs）纳入临床实践对于全面的疾病管理至关重要。
评估患者报告结局测量信息系统（PROMIS）工具在儿科克罗恩病（CD）患者中的反应性和临床实用性，并与标准临床评估工具进行比较。
一项纵向前瞻性研究，从儿科胃肠病科招募了31名年龄在8至17岁的儿科克罗恩病（CD）患者。在基线时以及18个月内每6个月收集一次数据。PROMIS儿科测量工具评估PROs。使用儿科克罗恩病活动指数（PCDAI）和临床标志物评估疾病活动度。还应用了IMPACT-III。采用线性混合效应模型（LMMs）和双变量分析来评估随时间的变化。
PROMIS全球健康评分随时间显示出显著改善，表明患者对整体健康的认知增强。PROMIS疼痛干扰和疲劳评分显著降低，表明身体健康状况改善。PROMIS抑郁评分总体下降，表明心理健康状况改善。PCDAI评分、血红蛋白和血小板计数显著变化，并与PROMIS测量结果相关。总体而言，该研究表明多个PROMIS测量指标发生了显著且具有临床意义的变化，证实了它们对疾病活动度变化的反应性。
PROMIS工具在儿科CD的管理中具有临床实用性，能为全球健康和生活质量提供有价值的见解。将PROMIS测量指标纳入常规临床实践可能会加强儿科IBD患者的疾病管理和治疗策略。
