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Author Notes:

Julie A. Panepinto, Phone: 414-955-4170, Email: jpanepin@mcw.edu

JP, DB, LC, and CC made substantial contributions to the conception, design, and acquisition of data and analysis and interpretation of data.

JP was responsible for drafting the manuscript.

All authors have been involved in acquisition of the data and in revising the manuscript critically and have given final approval of the version to be published and agree to be accountable for all aspects of the work.

We would like to thank the following research staff for their work on the project: Mark Nimmer, Sylvia Torres, and Duke Wagner, Medical College of Wisconsin, Milwaukee, WI;, Joanna Westerfield, Children’s National Medical Center, Washington, DC; I; Jason Czachor, Wayne State University/Children’s Hospital of Michigan, Detroit, MI; Laura Turner, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; Ashley Woodford, Children’s Hospital of Philadelphia, Philadelphia, PA; Kathleen Calabro, Children’s Hospital of Pittsburgh of UPMC, Pittsburgh, PA; Karina Soto-Ruiz and Victor Gonzalez, Baylor College of Medicine/Texas Children’s Hospital, Houston, TX; Virginia Koors, Washington University School of Medicine, St. Louis, MO; Marie Kay, Heather Gramse, Sally Jo Zuspan, Casey Evans, Jun Wang, Tim Simmons and Angie Webster, University of Utah/Data Coordinating Center, Salt Lake City, UT.

This concept and proposal was approved by the members of the PECARN Steering Committee, and all work was reviewed by the Data Coordinating Center, and the PECARN subcommittees: Grants and Publications, Protocol Review and Development, Feasibility and Budget and Quality Assurance.

Finally, we thank those who served on the Data Safety and Monitoring Board: Kathleen Neville MD, MS; Maria Mori Brooks PhD; Walt Schalick, III MD, PhD; Cage Johnson MD; Lalit Bajaj MD, MPH; David Schoenfeld MA, PhD.

This information or content and conclusions are those of the author and should not be construed as the official position or policy of, nor should any endorsements be inferred by HRSA, Department of Health and Human Services or the United States Government.

The authors declare that they have no competing interests.

Subjects:

Research Funding:

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:

  • Science & Technology
  • Life Sciences & Biomedicine
  • Health Care Sciences & Services
  • Health Policy & Services
  • Sickle cell disease
  • Quality of life
  • Acute pain crises
  • Longitudinal validity
  • Responsiveness
  • QUALITY-OF-LIFE
  • PATIENT-REPORTED OUTCOMES
  • RANDOMIZED CONTROLLED-TRIAL
  • INTRAINDIVIDUAL CHANGES
  • INTRAVENOUS MAGNESIUM
  • CHILDREN
  • RESPONSIVENESS
  • RELIABILITY
  • CRISIS

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

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Journal Title:

Health and Quality of Life Outcomes

Volume:

Volume 15, Number 1

Publisher:

, Pages 124-124

Type of Work:

Article | Final Publisher PDF

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

Copyright information:

© 2017 The Author(s).

This is an Open Access work distributed under the terms of the Creative Commons Attribution 4.0 International License (https://creativecommons.org/licenses/by/4.0/).
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