Matsumoto Hiroko, Hyman Joshua E, Shah Hitesh H, Sankar Wudbhav N, Laine Jennifer C, Mehlman Charles T, Schrader Tim, Kelly Derek M, Rosenfeld Scott B, Janicki Joseph A, Thacker Mihir M, Trupia Evan, Mcguire Molly F, Kim Harry K W
Department of Orthopaedic Surgery, Division of Pediatric Orthopaedic Surgery, Columbia University Medical Center.
Department of Epidemiology, Mailman School of Public Health, Columbia University, New York, NY.
J Pediatr Orthop. 2020 May-Jun;40(5):235-240. doi: 10.1097/BPO.0000000000001423.
Patient-reported outcomes (PRO) assessing health-related quality of life (HRQoL) are important outcome measures, especially in Legg-Calvé-Perthes disease (LCPD) where symptoms (pain and limping), activity restrictions, and treatments vary depending on the stage of the disease. The purpose of this study was to investigate the validity of the Patient-reported Outcomes Measurement Information System (PROMIS) for measuring HRQoL of patients with LCPD in various stages of the disease.
This is a multicenter validity study. Patients with LCPD between 4 and 18 years old were included and classified into modified Waldenström stages of disease: Early (1 or 2A), Late (2B or 3), or Healed (4). Seven PROMIS domains were collected, including Pain Interference, Fatigue, Mobility, Depression, Anger, Anxiety, and Peer Relationships. Convergent, discriminant, and known group validity was determined.
A total of 190 patients were included (mean age: 10.4±3.1 y). All 7 domains showed the worst scores in patients in the Early stage (known group validity). Within each domain, all domains positively correlated to each other (convergent validity). Patients who reported more anxiety, depression, and anger were associated with decreased mobility and increased fatigue and pain. Peer relationships had no to weak associations with other domains (discriminant validity).
PROMIS has construct validity in measuring the HRQoL of patients in different stages of LCPD, suggesting that PROMIS has potential to serve as a patient-reported outcome tool for this population.
Diagnostic level III study.
患者报告结局（PRO）评估与健康相关的生活质量（HRQoL）是重要的结局指标，尤其是在Legg-Calvé-Perthes病（LCPD）中，其症状（疼痛和跛行）、活动限制以及治疗方法会因疾病阶段而异。本研究的目的是调查患者报告结局测量信息系统（PROMIS）在测量LCPD不同疾病阶段患者的HRQoL方面的有效性。
这是一项多中心有效性研究。纳入4至18岁的LCPD患者，并将其分为改良的Waldenström疾病阶段：早期（1或2A）、晚期（2B或3）或愈合期（4）。收集了7个PROMIS领域的数据，包括疼痛干扰、疲劳、活动能力、抑郁、愤怒、焦虑和同伴关系。确定了收敛效度、区分效度和已知组效度。
共纳入190例患者（平均年龄：10.4±3.1岁）。所有7个领域在早期患者中得分最差（已知组效度）。在每个领域内，所有领域之间均呈正相关（收敛效度）。报告更多焦虑、抑郁和愤怒的患者与活动能力下降、疲劳和疼痛增加有关。同伴关系与其他领域的关联不显著或较弱（区分效度）。
PROMIS在测量LCPD不同阶段患者的HRQoL方面具有结构效度，这表明PROMIS有潜力作为该人群的患者报告结局工具。
诊断性III级研究。
