Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL (TM) Sickle Cell Disease Module

• Type of Material: Article
• Authors: Julie A. Panepinto, Medical College of Wisconsin; J. Paul Scott, Medical College of Wisconsin; Oluwakemi Badaki-Makun, Johns Hopkins University; Deepika S. Darbari, Childrens National Medical Center; Corrie E. Chumpitazi, Baylor College of Medicine; Gladstone E. Airewele, Baylor College of Medicine; Angela M. Ellison, Childrens Hospital of Philadelphia; Kim Smith-Whitley, Childrens Hospital of Philadelphia; Prashant Mahajan, Wayne State University; Sharada A. Sarnaik, Wayne State University; T Charles Casper, University of Utah; Larry J. Cook, University of Utah; Julie Leonard, Childrens National Medical Center; Monica L. Hulbert, Washington University; Elizabeth C. Powell, Northwestern University; Robert I. Liem, Northwestern University Feinberg School of Medicine; Robert Hickey, University of Pittsburgh; Lakshmanan Krishnamurti, Emory University; Cheryl A. Hillery, University of Pittsburgh; David C. Brousseau, Children’s Hospital of Wisconsin
• Language: English
• Date: 2017-06-12
• Publisher: BioMed Central
• Publication Version: Final Published Version
• Copyright Statement: © 2017 The Author(s).
• License: Creative Commons Attribution 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1186/s12955-017-0700-2
• Title of Journal or Parent Work: Health and Quality of Life Outcomes
• ISSN: 1477-7525
• Volume: 15
• Issue: 1
• Start Page: 124
• End Page: 124
• Grant/Funding Information: National Heart, Lung, and Blood Institute of the National Institutes of Health under Award Number 1R01HL103427-01A1 (J.P.).; Eunice Kennedy Shriver National Institute of Child Health & Human Development under Award Number 5R01HD062347–01 and Administrative Supplement Number 3R01HD062347-03S1 (D.B.).; This project is supported in part by the Health Resources and Services Administration (HRSA), Maternal and Child Health Bureau (MCHB), Emergency Medical Services for Children (EMSC) Network Development Demonstration Program under cooperative agreement number U03MC00008, and is partially supported by MCHB cooperative agreements: U03MC00001, U03MC00003, U03MC00006, U03MC00007, U03MC22684, U03MC22685.
• Keywords: Acute pain crises; Health Care Sciences & Services; Life Sciences & Biomedicine; INTRAVENOUS MAGNESIUM; QUALITY-OF-LIFE; RELIABILITY; Sickle cell disease; RANDOMIZED CONTROLLED-TRIAL; Health Policy & Services; CRISIS; CHILDREN; RESPONSIVENESS; Quality of life; Longitudinal validity; INTRAINDIVIDUAL CHANGES; Science & Technology; Responsiveness; PATIENT-REPORTED OUTCOMES
• Research Categories: Health Sciences, Oncology; Health Sciences, Human Development

Abstract

Background: 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. Methods: 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. Results: 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. Conclusions: 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. Trial Registration: ClinicalTrials.gov (study identifier: NCT01197417 ). Date of registration: 08/30/2010