Evaluating anchor variables and variation in meaningful score differences for PROMIS® Pediatric measures in children and adolescents living with a rheumatic disease.

IF 3.3 3区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Quality of Life Research Pub Date : 2024-10-14 DOI:10.1007/s11136-024-03800-2
C K Zigler, Z Li, A Hernandez, R L Randell, C M Mann, E Weitzman, L E Schanberg, E von Scheven, B B Reeve
{"title":"Evaluating anchor variables and variation in meaningful score differences for PROMIS<sup>®</sup> Pediatric measures in children and adolescents living with a rheumatic disease.","authors":"C K Zigler, Z Li, A Hernandez, R L Randell, C M Mann, E Weitzman, L E Schanberg, E von Scheven, B B Reeve","doi":"10.1007/s11136-024-03800-2","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose: </strong>Meaningful score differences (MSDs), as defined by recent FDA guidance, can improve the interpretation of outcome measure scores and score changes. Well-accepted methods for estimating MSDs typically rely on external anchor variables, but the applications of these methods are limited in children and adolescents with rheumatic diseases. This project explored multiple candidate anchors for the PROMIS<sup>®</sup> Pediatric measures of Physical Activity, Fatigue, Pain Interference, and Mobility for children with juvenile idiopathic arthritis (JIA) or systemic lupus erythematosus (SLE).</p><p><strong>Methods: </strong>Longitudinal data were extracted from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. Candidate anchors included patient-reported domain-specific global impressions of change (GIC) along with other parent- and clinician-reported variables. Prior to MSD estimation, the quality of the anchors was assessed using a priori criteria (correlation ≥0.30, n≥10, <10% missing). Anchors meeting criteria were used to calculate MSDs.</p><p><strong>Results: </strong>Among 289 children with JIA and 47 with SLE, the GIC did not meet criteria inhalf of the scenarios. Other candidate anchors performed slightly better. The calculated MSDs varied by external anchor across measures, diagnoses, and direction of change (better vs worse).</p><p><strong>Conclusions: </strong>Many of the candidate external anchoring variables did not meet pre-specified criteria for calculating MSDs. Even for those that did, the choice of anchoring variable had a strong impact on the estimated MSD value and were different from other published values. As in adults, establishing pediatric MSDs requires selection of high-quality anchors, as changes in the variables used as anchors can impact MSD values and any subsequent score interpretations.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3000,"publicationDate":"2024-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Quality of Life Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s11136-024-03800-2","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0

Abstract

Purpose: Meaningful score differences (MSDs), as defined by recent FDA guidance, can improve the interpretation of outcome measure scores and score changes. Well-accepted methods for estimating MSDs typically rely on external anchor variables, but the applications of these methods are limited in children and adolescents with rheumatic diseases. This project explored multiple candidate anchors for the PROMIS® Pediatric measures of Physical Activity, Fatigue, Pain Interference, and Mobility for children with juvenile idiopathic arthritis (JIA) or systemic lupus erythematosus (SLE).

Methods: Longitudinal data were extracted from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry. Candidate anchors included patient-reported domain-specific global impressions of change (GIC) along with other parent- and clinician-reported variables. Prior to MSD estimation, the quality of the anchors was assessed using a priori criteria (correlation ≥0.30, n≥10, <10% missing). Anchors meeting criteria were used to calculate MSDs.

Results: Among 289 children with JIA and 47 with SLE, the GIC did not meet criteria inhalf of the scenarios. Other candidate anchors performed slightly better. The calculated MSDs varied by external anchor across measures, diagnoses, and direction of change (better vs worse).

Conclusions: Many of the candidate external anchoring variables did not meet pre-specified criteria for calculating MSDs. Even for those that did, the choice of anchoring variable had a strong impact on the estimated MSD value and were different from other published values. As in adults, establishing pediatric MSDs requires selection of high-quality anchors, as changes in the variables used as anchors can impact MSD values and any subsequent score interpretations.

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
评估风湿病儿童和青少年 PROMIS® 儿科测量的锚变量和有意义分数差异的变化。
目的:根据美国食品药品管理局(FDA)最新指南的定义,有意义分数差异(MSDs)可以改善对结果测量分数和分数变化的解释。公认的估算 MSD 的方法通常依赖于外部锚变量,但这些方法在患有风湿病的儿童和青少年中的应用有限。本项目针对幼年特发性关节炎(JIA)或系统性红斑狼疮(SLE)患儿,探索了 PROMIS® 儿科体力活动、疲劳、疼痛干扰和活动能力测量的多个候选锚变量:方法:从儿童关节炎和风湿病学研究联盟(CARRA)登记处提取纵向数据。候选锚点包括患者报告的特定领域总体变化印象(GIC)以及其他家长和临床医生报告的变量。在估算 MSD 之前,使用先验标准评估了锚点的质量(相关性≥0.30,n≥10,结果):在289名JIA患儿和47名系统性红斑狼疮患儿中,GIC在半数情况下不符合标准。其他候选锚点的表现稍好。不同的外部锚点在测量、诊断和变化方向(更好与更差)上计算出的MSD各不相同:结论:许多候选外部锚变量都不符合计算 MSD 的预设标准。结论:许多候选外部锚定变量不符合计算 MSD 的预先指定标准,即使符合标准,锚定变量的选择对估计 MSD 值也有很大影响,并且与其他已公布的值不同。与成人一样,确定儿科 MSD 值需要选择高质量的锚定变量,因为用作锚定变量的变量变化会影响 MSD 值和任何后续评分解释。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
Quality of Life Research
Quality of Life Research 医学-公共卫生、环境卫生与职业卫生
CiteScore
6.50
自引率
8.60%
发文量
224
审稿时长
3-8 weeks
期刊介绍: Quality of Life Research is an international, multidisciplinary journal devoted to the rapid communication of original research, theoretical articles and methodological reports related to the field of quality of life, in all the health sciences. The journal also offers editorials, literature, book and software reviews, correspondence and abstracts of conferences. Quality of life has become a prominent issue in biometry, philosophy, social science, clinical medicine, health services and outcomes research. The journal''s scope reflects the wide application of quality of life assessment and research in the biological and social sciences. All original work is subject to peer review for originality, scientific quality and relevance to a broad readership. This is an official journal of the International Society of Quality of Life Research.
期刊最新文献
Health-related quality of life due to malaria: a systematic review. Identification of meaningful individual-level change thresholds for worsening on the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE®). Symptom prevalence in patients with advanced heart failure and its association with quality of life and activities of daily living. Longitudinal validation of the PROMIS-16 in a sample of adults in the United States with back pain. Norwegian and Swedish value sets for the EORTC QLU-C10D utility instrument.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1