Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module.
Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module.
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DOI：
10.1186/s12955-017-0700-2
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发表时间：
2017-06-12
影响因子：
3.6
通讯作者：
Pediatric Emergency Care Applied Research Network (PECARN)
中科院分区：
文献类型：
--
作者：
Panepinto JA;Paul Scott J;Badaki-Makun O;Darbari DS;Chumpitazi CE;Airewele GE;Ellison AM;Smith-Whitley K;Mahajan P;Sarnaik SA;Charles Casper T;Cook LJ;Leonard J;Hulbert ML;Powell EC;Liem RI;Hickey R;Krishnamurti L;Hillery CA;Brousseau DC;Pediatric Emergency Care Applied Research Network (PECARN)
使用健康相关生活质量 (HRQL) 工具来衡量以患者为中心的结果时，检测健康状况随时间的变化并确定有意义的变化是关键要素。 PedsQL™ 镰状细胞病模块是一种疾病特异性 HRQL 仪器，此前已被证明是有效且可靠的。我们的目标是确定 PedsQL™ 镰状细胞病模块的纵向有效性以及对患者有意义的 HRQL 变化。利用多中心前瞻性试验设计进行了一项辅助研究。因急性疼痛性血管闭塞危象而入院治疗的 4 至 21 岁镰状细胞病儿童符合资格。儿童在三个时间点完成 HRQL 评估（在急诊室、出院后一周和返回基线时（出院后一到三个月）。主要结局是 HRQL 评分的变化。使用基于分布（效应大小、测量标准误差 (SEM)）和锚定（整体变化评估）的方法来确定 HRQL 的纵向有效性和有意义的变化。通过将变化分数锚定到患者对疼痛整体改善的感知，确定对患者有意义的 HRQL 变化。中等效果除沟通 I 和认知疲劳领域外，所有领域均确定了 1 SEM 的大小（0.20-0.80），超过 50% 的患者在总 HRQL 评分上改善了至少 1 SEM。HRQL 变化评分为 7-10 表示 HRQL 的感知改善最小，HRQL 变化评分为 18 或更高表示中度至较大改善。 PedsQL™ 镰状细胞疾病模块对经历急性疼痛性血管闭塞危机的患者的 HRQL 变化做出反应。该研究数据为 PedsQL™ 镰状细胞疾病模块建立了纵向有效性和有意义的变化参数（研究编号：NCT01197417）。本文的在线版本。 (doi:10.1186/s12955-017-0700-2) 包含可供授权用户使用的补充材料。
Detecting change in health status over time and ascertaining meaningful changes are critical elements when using health-related quality of life (HRQL) instruments to measure patient-centered outcomes. The PedsQL™ Sickle Cell Disease module, a disease specific HRQL instrument, has previously been shown to be valid and reliable. Our objectives were to determine the longitudinal validity of the PedsQL™ Sickle Cell Disease module and the change in HRQL that is meaningful to patients. An ancillary study was conducted utilizing a multi-center prospective trial design. Children ages 4–21 years with sickle cell disease admitted to the hospital for an acute painful vaso-oclusive crisis were eligible. Children completed HRQL assessments at three time points (in the Emergency Department, one week post-discharge, and at return to baseline (One to three months post-discharge). The primary outcome was change in HRQL score. Both distribution (effect size, standard error of measurement (SEM)) and anchor (global change assessment) based methods were used to determine the longitudinal validity and meaningful change in HRQL. Changes in HRQL meaningful to patients were identified by anchoring the change scores to the patient’s perception of global improvement in pain. Moderate effect sizes (0.20–0.80) were determined for all domains except the Communication I and Cognitive Fatigue domains. The value of 1 SEM varied from 3.8–14.6 across all domains. Over 50% of patients improved by at least 1 SEM in Total HRQL score. A HRQL change score of 7–10 in the pain domains represented minimal perceived improvement in HRQL and a HRQL change score of 18 or greater represented moderate to large improvement. The PedsQL™ Sickle Cell Disease Module is responsive to changes in HRQL in patients experiencing acute painful vaso-occlusive crises. The study data establish longitudinal validity and meaningful change parameters for the PedsQL™ Sickle Cell Disease Module. ClinicalTrials.gov (study identifier: NCT01197417). Date of registration: 08/30/2010 The online version of this article (doi:10.1186/s12955-017-0700-2) contains supplementary material, which is available to authorized users.