Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force

Matza, Louis S.; Patrick, Donald L.; Riley, Anne W.; Alexander, John J.; Rajmil, Luís; Pleil, Andreas M.; Bullinger, Monika

doi:10.1016/j.jval.2013.04.004

Cited by 363 publications

(511 citation statements)

References 130 publications

(200 reference statements)

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“…The latter are therefore the method of choice in clinical trials (Walterfang et al 2013). Since the concept of HrQoL is based on subjective experience, self-assessment using ageappropriate instruments is the gold standard (Matza et al 2013). Self and proxy reports are not interchangeable (Upton et al 2008), but proxy reports are valuable in very young or cognitively impaired patients.…”

Section: Introductionmentioning

confidence: 99%

“…In this process, patients and parents are involved as "content experts" (Matza et al 2004). Focus group interviews are the method of choice to identify topics, concerns and resources relevant in everyday life (Matza et al 2013). This paper presents the qualitative content analysis of focus group and single interviews with IT-IEM patients and their parents.…”

Section: Introductionmentioning

confidence: 99%

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Living with Intoxication-Type Inborn Errors of Metabolism: A Qualitative Analysis of Interviews with Paediatric Patients and Their Parents

et al. 2016

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Introduction: Progress in diagnosis and treatment of patients with intoxication-type inborn errors of metabolism (IT-IEM) such as urea cycle disorders, organic acidurias or maple syrup urine disease is resulting in a growing number of long-term survivors. Consequently, health-related quality of life (HrQoL) of patients is increasingly regarded as a meaningful outcome parameter. To develop the first validated, disease-specific HrQoL questionnaire for IT-IEM, patients and parents were interviewed as content experts to identify major physical and psychosocial constraints and resources.Methods: Focus group interviews with 19 paediatric IT-IEM patients and 26 parents were conducted in four metabolic centres in Austria, Germany and Switzerland. Disease-specific HrQoL categories were established by qualitative content analysis.Results: Fourteen disease-specific topics related to the three well-established generic HrQoL dimensions of physi-

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Living with Intoxication-Type Inborn Errors of Metabolism: A Qualitative Analysis of Interviews with Paediatric Patients and Their Parents

et al. 2016

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“…Our findings informed modifications to every SCIM-III SR item and response scale, producing a pediatric version of the tool that we 18 assuming that the items and response scales are written with clarity, simplicity and minimal cognitive burden. 19 We previously showed that youths with SCI as young as age 8 were able to understand and respond to questions. 13 A fundamental problem with the SCIM-III SR was that youths did not understand the majority of the words and phrases.…”

Section: Discussionmentioning

confidence: 99%

“…Engaging children to evaluate reading and comprehension of items on patient reported outcome instruments, as has been done in this study, is essential prior to using them in research or practice. 19 Two other problems encountered by children when reading and responding to the SCIM-III SR were vagueness of terminology and unfamiliarity with medical jargon. Even though the youths in this study had been injured for approximately 4½ years, they were unfamiliar with the meaning of the medical words in the SCIM-III SR. As a way to eliminate medical jargon and provide clarity, preambles enabled children to understand and appropriately respond to items that asked about complex health situations.…”

Section: Discussionmentioning

confidence: 99%

“…On the basis of our prior work 13,14 and published literature, 19 we estimated that we would need to perform cognitive testing interviews with at least five subjects of various ages to understand how children of different ages responded to the SCIM-III SR. In this study, the same words and phrases were repeatedly problematic, and youths' suggestions for change were relatively consistent across the ages.…”

Section: Discussionmentioning

confidence: 99%

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The spinal cord independence measure (SCIM)-III self report for youth

et al. 2015

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Study design: The items and response scales of the Spinal Cord Independence Measure (SCIM-III) self report (SR) were exposed to formal cognitive testing with children with SCI, and in parallel a survey using the modified Delphi Technique was conducted to engage content experts in an iterative critical review of the SCIM-III SR. Objectives: To evaluate the validity of the SCIM-III SR for pediatric utilization. Setting: United States of America. Methods: Formal cognitive testing was conducted with 17 youths with SCI and required them to read and answer each SCIM-III SR item aloud. Think aloud methodology was used to record details about how youths understood and interpreted items and why they selected a given response. The interviews were audiotaped and transcribed verbatim. Content analysis focused on identifying words that children could not read or understand. In parallel, the Modified Delphi Technique engaged expert therapists to critically review the SCIM-III SR for pediatric utilization. Results: Every SCIM-III SR item and response scale required modification before children were able to read, understand and respond to them. Youth encountered difficulties because of vague terms, medical jargon and complex words and phrases. Three iterative Delphi rounds were required before achieving 80% agreement that items and response scales were written well for children. Conclusion: Our findings informed modifications to every SCIM-III SR item and response scale, producing a pediatric version of the tool that we formally refer to as the SCIM-III SR-Youth (SCIM-III SR-Y).

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Validity and Reliability of Four Parent/Patient–Reported Outcome Measures for Juvenile Idiopathic Arthritis Remote Monitoring

Dijkhuizen

Ridella

Naddei

et al. 2022

Arthritis Care & Research

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Objective The aim of this work was to provide evidence of validity and reliability for 4 parent/child–reported outcome measures included in the Outcome Measures in Rheumatology juvenile idiopathic arthritis core domain set: the evaluation of the child's pain and level of disease activity, the assessment of morning stiffness duration, and an active joint count for proxy/self‐assessment. Methods Patients were included in the multinational study Epidemiology Treatment and Outcome of Childhood Arthritis. Criterion validity was assessed by examining the correlation of the 4 tested measures with physician measures and the clinical Juvenile Arthritis Disease Activity Score in 10 joints (cJADAS10) in the whole sample and after grouping patients by International League of Associations for Rheumatology (ILAR) category, geographic area, and education level. Reliability was assessed comparing 2 visits 7–14 days apart with intraclass correlation coefficients (ICCs). Results A total of 8,643 parents and 6,060 patients had all the evaluations available. Correlations of tested measures were moderate (0.4–0.7) with physician‐reported measures. The level of correlation with the cJADAS10 remained stable after grouping patients by ILAR category, geographic areas, and level of education of the parent filling the questionnaire. In 442 parents and 344 children, ICCs ranged between 0.79 and 0.87 for parents and 0.81 and 0.88 for children. Conclusion The 4 tested parent/child–reported outcomes showed good criterion validity and excellent reliability. These tools can be considered for remote patient assessment, when in‐person evaluation might not be possible.

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Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force

Cited by 363 publications

References 130 publications

Living with Intoxication-Type Inborn Errors of Metabolism: A Qualitative Analysis of Interviews with Paediatric Patients and Their Parents

Living with Intoxication-Type Inborn Errors of Metabolism: A Qualitative Analysis of Interviews with Paediatric Patients and Their Parents

The spinal cord independence measure (SCIM)-III self report for youth

Validity and Reliability of Four Parent/Patient–Reported Outcome Measures for Juvenile Idiopathic Arthritis Remote Monitoring

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