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Evaluation of health related quality of life in children with immune thrombocytopenia with the PedsQL™ 4.0 Generic Core Scales: a study on behalf of the pays de Loire pediatric hematology network.

ABSTRACT: Immune thrombocytopenia (ITP) is a childhood disorder that is often life-altering for children and their parents. Health related quality of life (HRQL) has never been chronologically monitored in children with ITP. We initiated a prospective study to assess HRQL from diagnosis to six months and define factors that influence this outcome in children with ITP.73 children with acute ITP aged from 2 to 18 years were prospectively enrolled in the study. According to the presence of bleeding, they were or were not given a 4-day course of corticosteroid treatment. The PedsQL™ 4.0 Generic Core Scale was completed by children and parents upon their inclusion in the study and 6 months after diagnosis.Over the six month period, quality of life improved in terms of their global, physical and psychosocial well-being for 54.5%, 35.6% and 36.2% of patients respectively. This improvement is clinically relevant compared to scores at diagnosis, corresponding at least to a minimal clinically important difference (MCID). Factors such as sex, age, platelet count, bleeding scores, bone marrow aspiration and persistence of ITP at 6 months were not significantly associated with HRQL scores. However, preceding viral infection was identified to have an impact on HRQL.This first longitudinal study assessing HRQL in children with ITP reveals a global improvement in PedSQL™ 4.0. However, these results should be considered with caution since our data also confirm that self-report HRQL scores are not influenced by any analyzed biologic or clinical parameters. Others tools, such as Kids' ITP Tools, would probably be required to assess the HRQL of this population.Trial registration clinical trials.gov Identifier: NCT00331357.

SUBMITTER: Strullu M

PROVIDER: S-EPMC3830442 | biostudies-literature | 2013 Nov

REPOSITORIES: biostudies-literature

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Evaluation of health related quality of life in children with immune thrombocytopenia with the PedsQL™ 4.0 Generic Core Scales: a study on behalf of the pays de Loire pediatric hematology network.

Strullu Marion M Rakotonjanahary Josué J Tarral Eliane E Savagner Christophe C Thomas Caroline C Méchinaud Françoise F Reguerre Yves Y Poignant Sylvaine S Boutet Arnaud A Bassil Joachim J Médinger Dominique D Quemener Emmanuel E Young Nancy L NL Rachieru Petronela P Klaassen Robert J RJ Pellier Isabelle I

Health and quality of life outcomes 20131113

<h4>Background</h4>Immune thrombocytopenia (ITP) is a childhood disorder that is often life-altering for children and their parents. Health related quality of life (HRQL) has never been chronologically monitored in children with ITP. We initiated a prospective study to assess HRQL from diagnosis to six months and define factors that influence this outcome in children with ITP.<h4>Methods</h4>73 children with acute ITP aged from 2 to 18 years were prospectively enrolled in the study. According to ...[more]

PMID: 24225051

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Project description:BackgroundThe Pediatric Quality of Life Inventory Version 4.0 (PedsQLTM4.0) is a generic health-related quality of life (HRQoL) questionnaire, widely used in pediatric clinical trials but not yet validated in France. We performed the psychometric validation of the self and proxy PedsQLTM4.0 generic questionnaires for French children aged 8-12 years old.MethodsThis bicentric cross-sectional study included 123 children and their parents with congenital heart disease (CHD) and 97 controls. The psychometric validation method was based on the consensus-based standards for the selection of health measurement instruments (COSMIN). The reliability was tested using the intraclass correlation coefficient (ICC). To evaluate the validity of this scale, content, face, criterion, and construct validity psychometric proprieties were tested. Acceptability was studied regarding questionnaires' completion and the existence of a floor or a ceiling effect.ResultsTest-retest reliability intra-class correlation coefficients were mainly in good range (0.49-0.66). Face validity was very good among parents (0.85) and children (0.75). Content validity was good (0.70), despite misinterpretation of some items. In construct validity, each subscale had acceptable internal consistency reliability (Cronbach's ??>?0.72 in self-reports,?>?0.69 in proxy-reports). In the confirmatory factor analysis, the goodness-of-fit statistics rejected the original structure with 4 factors. The exploratory factor analysis revealed an alternative two-factor structure corresponding to physical and psychological dimensions. Convergent validity was supported by moderate (>?0.41) to high correlations (0.57) between PedsQL and Kidscreeen questionnaires for physical, emotion and school dimensions. The ability of the PedsQL to discriminate CHD severity was better with physical, social and total scores for both self-reports and proxy-reports.ConclusionsThe PedsQLTM4.0 generic self and proxy HRQoL questionnaires found good psychometric properties, with regard to acceptability, responsiveness, validity, and reliability. This instrument appeared to be easy to use and comprehend within the target population of children aged 8 to 12 years old and their parents.Trial registrationThis study was approved by the South-Mediterranean-IV Ethics Committee and registered on ClinicalTrials.gov (NCT01202916), https://clinicaltrials.gov/ct2/show/NCT01202916 .

Project description:BackgroundThe Pediatric Quality of Life™ 3.0 Neuromuscular Module is an instrument to assess health-related quality of life (HRQoL) among children with neuromuscular diseases (NMDs) aged 2-18 years. This study aimed to determine whether the PedsQL™ 3.0 Neuromuscular Module Indonesian Version is valid and reliable.MethodsThis study used the Indonesian translation of the PedsQL™ 3.0 Neuromuscular Module after getting formal permission from the inventor, and the translation process followed the Mapi linguistic translation guidelines. This study administered the PedsQL™ 3.0 Neuromuscular Module Indonesian Version to 84 parents and 71 children. In addition, we used the Rasch model to analyze the psychometric properties.ResultsThe reliability of the total scale of the PedsQL™ Neuromuscular Module Indonesian Version shows good to very good criteria. On the parent proxy-report, Cronbach alpha was 0.95, person reliability was 0.84, item reliability was 0.93, person separation was 2.32, item separation was 3.61, person strata separation was 4 levels, and item strata separation was 5 levels. On the child self-report, Cronbach alpha was 0.93, person reliability was 0.81, item reliability was 0.81, person separation was 2.08, item separation was 2.06, person strata separation was 3 levels, and item strata separation was 3 levels. The total scale of the PedsQL Neuromuscular Module Indonesian Version shows fair to good construct validity in parent proxy-report (explained variance 51.9%; unexplained variance 8.4%) and child self-report (explained variance 40.9%; unexplained variance 12.6%). There were no misfit items in the parent proxy-report (infit 0.66-1.49; outfit 0.51-1.81; point measure correlation 0.36-0.93) and child self-report (infit 0.53-1.65; outfit 0.50-1.73; point measure correlation 0.31-0.90) identified by the Rasch models.ConclusionsThe PedsQL™ 3.0 Neuromuscular Modul Indonesian Version is a valid and reliable instrument in measuring HRQoL in Indonesian children with neuromuscular diseases.

Project description:BackgroundDetecting 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.MethodsAn 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.ResultsModerate 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.ConclusionsThe 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 registrationClinicalTrials.gov (study identifier: NCT01197417 ). Date of registration: 08/30/2010.

Dataset Information

Evaluation of health related quality of life in children with immune thrombocytopenia with the PedsQL™ 4.0 Generic Core Scales: a study on behalf of the pays de Loire pediatric hematology network.

Publications

Evaluation of health related quality of life in children with immune thrombocytopenia with the PedsQL™ 4.0 Generic Core Scales: a study on behalf of the pays de Loire pediatric hematology network.

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