Azevedo Sara, Oliveira Maria Miguel, Nogueira Paulo, Lopes Ana Isabel
Gastroenterology Unit, Pediatric Department, Santa Maria University Hospital - CHLN, Academic Medical Centre of Lisbon, Lisbon, Portugal.
Medical School, University of Lisbon, Lisbon, Portugal.
Health Qual Life Outcomes. 2024 Dec 31;22(1):112. doi: 10.1186/s12955-024-02330-2.
This study evaluated the clinical utility of the Patient-Reported Outcomes Measurement Information System (PROMIS) by comparing it with objective clinical data and validated health-related quality of life (HRQOL) measures in pediatric Crohn's disease (CD) patients.
Cross-sectional study. Pediatric CD patients (aged 8-17 years) were enrolled prospectively over eight months from an outpatient pediatric gastroenterology center. We assessed the associations between PROMIS Pediatric short-form measures, demographic and disease-related data, global clinical assessments, and HRQOL measures. A subanalysis according to the PCDAI (remission versus active disease) was also conducted.
Thirty-one patients (mean age: 15.3; 58% female) with a mean disease duration of 2.7 years were included; 80.6% were in remission or had mild disease. The PROMIS score was significantly correlated with several factors: age was negatively correlated with the PROMIS Global Health Scale (r=-0.399; p = 0.026) and Life Satisfaction (r=-0.359; p = 0.047); sex was associated with the PROMIS Cognitive Function Scale (t = 2.20; p = 0.038), favoring males; and school level was inversely related to the PROMIS Peer Relationships (F = 3.90; p = 0.003). Clinical assessments also revealed significant correlations between hemoglobin and PROMIS Global Health (r = 0.356; p = 0.049) and pain interference (r=-0.360; p = 0.046) and between ferritin and PROMIS Meaning and Purpose (r = 0.435; p = 0.016) and cognitive function (r = 0.450; p = 0.011). Disease activity assessments correlated significantly with multiple PROMIS measures, with better scores in patients in remission. Treatment changes, particularly corticosteroid treatment, negatively impacted the PROMIS Anxiety and Life Satisfaction scores. IMPACT-III scores correlated positively with PROMIS Global Health, Meaning and Purpose, Life Satisfaction, and peer relationships scores and negatively with Depression, Anxiety, Pain interference, and Fatigue scores. Group analysis indicated better PROMIS scores and HRQOL scores in remission than in active disease remission.
Consistent with recent evidence, PROMIS scores reliably reflect disease activity and HRQOL. The meaningful associations with clinical assessment and treatment efficacy reinforce the clinical relevance and utility of PROs in the patient-centered management of pediatric IBD and highlight the importance of self-reports as a gold standard tool for assessing health status.
本研究通过将患者报告结局测量信息系统（PROMIS）与儿科克罗恩病（CD）患者的客观临床数据及经过验证的健康相关生活质量（HRQOL）测量指标进行比较，评估其临床实用性。
横断面研究。在8个月的时间里，从一家儿科胃肠病门诊中心前瞻性纳入8至17岁的儿科CD患者。我们评估了PROMIS儿科简表测量指标、人口统计学和疾病相关数据、整体临床评估以及HRQOL测量指标之间的关联。还根据儿童克罗恩病活动指数（PCDAI，缓解期与活动期疾病）进行了亚分析。
纳入了31例患者（平均年龄：15.3岁；58%为女性），平均病程2.7年；80.6%处于缓解期或患有轻度疾病。PROMIS评分与多个因素显著相关：年龄与PROMIS全球健康量表呈负相关（r = -0.399；p = 0.026）以及生活满意度呈负相关（r = -0.359；p = 0.047）；性别与PROMIS认知功能量表相关（t = 2.20；p = 0.038），男性得分更高；学校水平与PROMIS同伴关系呈负相关（F = 3.90；p = 0.003）。临床评估还显示血红蛋白与PROMIS全球健康（r = 0.356；p = 0.049）和疼痛干扰（r = -0.360；p = 0.046）之间以及铁蛋白与PROMIS意义与目的（r = 0.435；p = 0.016）和认知功能（r = 0.450；p = 0.011）之间存在显著相关性。疾病活动评估与多个PROMIS测量指标显著相关，缓解期患者得分更高。治疗变化，尤其是皮质类固醇治疗，对PROMIS焦虑和生活满意度得分产生负面影响。IMPACT - III评分与PROMIS全球健康、意义与目的、生活满意度和同伴关系评分呈正相关，与抑郁、焦虑、疼痛干扰和疲劳评分呈负相关。分组分析表明，缓解期的PROMIS评分和HRQOL评分优于活动期疾病缓解期。
与近期证据一致，PROMIS评分可靠地反映疾病活动和HRQOL。与临床评估和治疗效果的有意义关联强化了患者报告结局在以患者为中心的儿科炎症性肠病管理中的临床相关性和实用性，并突出了自我报告作为评估健康状况的金标准工具 的重要性。
