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

and for the Pediatric Emergency Care Applied Research Network (PECARN)

Research output: Contribution to journalArticle

11 Citations (Scopus)

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

Original languageEnglish (US)
Article number124
JournalHealth and Quality of Life Outcomes
Volume15
Issue number1
DOIs
StatePublished - Jun 12 2017

Fingerprint

Sickle Cell Anemia
Quality of Life
Pain
Health Status
Fatigue
Hospital Emergency Service
Communication

Keywords

  • Acute pain crises
  • Longitudinal validity
  • Quality of life
  • Responsiveness
  • Sickle cell disease

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health

Cite this

and for the Pediatric Emergency Care Applied Research Network (PECARN). / Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module. In: Health and Quality of Life Outcomes. 2017 ; Vol. 15, No. 1.
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title = "Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module",
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",
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Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module. / and for the Pediatric Emergency Care Applied Research Network (PECARN).

In: Health and Quality of Life Outcomes, Vol. 15, No. 1, 124, 12.06.2017.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module

AU - and for the Pediatric Emergency Care Applied Research Network (PECARN)

AU - Panepinto, Julie A.

AU - Paul Scott, J.

AU - Badaki-Makun, Oluwakemi

AU - Darbari, Deepika S.

AU - Chumpitazi, Corrie E.

AU - Airewele, Gladstone E.

AU - Ellison, Angela M.

AU - Smith-Whitley, Kim

AU - Mahajan, Prashant

AU - Sarnaik, Sharada A.

AU - Charles Casper, T.

AU - Cook, Larry J.

AU - Leonard, Julie

AU - Hulbert, Monica L.

AU - Powell, Elizabeth C

AU - Liem, Robert I

AU - Hickey, Robert

AU - Krishnamurti, Lakshmanan

AU - Hillery, Cheryl A.

AU - Brousseau, David C.

PY - 2017/6/12

Y1 - 2017/6/12

N2 - 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

AB - 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

KW - Acute pain crises

KW - Longitudinal validity

KW - Quality of life

KW - Responsiveness

KW - Sickle cell disease

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