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

Health and Quality of Life Outcomes

for the Pediatric Emergency Care Applied Research Network (PECARN)¹  Elizabeth C. Powell²  Robert I. Liem³  Corrie E. Chumpitazi⁴  Gladstone E. Airewele⁵  Angela M. Ellison⁶  Kim Smith-Whitley⁷  Robert Hickey⁸  Deepika S. Darbari⁹  Lakshmanan Krishnamurti^1,10  Cheryl A. Hillery^1,11  Oluwakemi Badaki-Makun^1,12  David C. Brousseau^1,13  J. Paul Scott^1,14  Julie A. Panepinto^1,14  Julie Leonard^1,15  Larry J. Cook^1,16  T Charles Casper^1,16  Monica L. Hulbert^1,17  Prashant Mahajan^1,18  Sharada A. Sarnaik^1,19 

[1]

[2]Ann & Robert H. Lurie Children’s Hospital of Chicago, Division of Emergency Medicine, Northwestern University Feinberg School of Medicine

[3]Ann & Robert H. Lurie Children’s Hospital of Chicago, Hematology, Oncology & Stem Cell Transplant

[4]Baylor College of Medicine/Texas Children’s Hospital, Pediatric Emergency Medicine

[5]Baylor College of Medicine/Texas Children’s Hospital, Pediatric Hematology and Oncology

[6]Children’s Hospital of Philadelphia, Pediatric Emergency Medicine

[7]Children’s Hospital of Philadelphia, Pediatric Hematology and Oncology

[8]Children’s Hospital of Pittsburgh of University of Pittsburgh Medical Center, Pediatric Emergency Medicine

[9]Children’s National Medical Center, Pediatric Hematology and Oncology

[10]Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University School of Medicine

[11]Department of Pediatrics, Division of Hematology/Oncology, University of Pittsburgh School of Medicine

[12]Johns Hopkins University, Pediatric Emergency Medicine

[13]Medical College of Wisconsin, Pediatric Emergency Medicine, and the Children’s Hospital of Wisconsin

[14]Medical College of Wisconsin, Pediatric Hematology and Oncology, and the Children’s Hospital of Wisconsin

[15]Nationwide Children’s Hospital, Pediatric Emergency Medicine

[16]University of Utah/Pediatric Emergency Care Applied Research Network Data Coordinating Center

[17]Washington University School of Medicine, Division of Pediatric Hematology and Oncology

[18]Wayne State University/Children’s Hospital of Michigan, Pediatric Emergency Medicine

[19]Wayne State University/Children’s Hospital of Michigan, Pediatric Hematology and Oncology

关键词: Sickle cell disease;    Quality of life;    Acute pain crises;    Longitudinal validity;    Responsiveness;   

DOI  :  10.1186/s12955-017-0700-2

来源: DOAJ

【 摘 要 】

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

【 授权许可】

Unknown