Engaging the Voices of Children: A Scoping Review of How Children and Adolescents Are Involved in the Development of Quality-of-Life–Related Measures

Willis, Jessica; Zeratkaar, Dena; Hove, Julia ten; Rosenbaum, Peter; Ronen, Gabriel M.

doi:10.1016/j.jval.2020.11.007

Cited by 10 publications

(15 citation statements)

References 130 publications

(172 reference statements)

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“…We expect to seek, engage with, and listen to the voices of the children and their parents who come to our clinics. 5 This way of working with patients has changed the nature of our clinical discourse. These are merely illustrations of the cultural (i.e.…”

Section: Childhood Disability: the Importance Of Contextmentioning

confidence: 99%

“…Examples include issues such as the meaning of disability (a social construct as conceptualized by WHO) versus impairment (a within‐the‐person problem); and an emphasis on functioning, however it is accomplished, as distinct from normal function. We expect to seek, engage with, and listen to the voices of the children and their parents who come to our clinics 5 . This way of working with patients has changed the nature of our clinical discourse.…”

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confidence: 99%

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Childhood disability: The importance of context

Rosenbaum¹

2022

Develop Med Child Neuro

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Section: Childhood Disability: the Importance Of Contextmentioning

confidence: 99%

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confidence: 99%

Childhood disability: The importance of context

Rosenbaum¹

2022

Develop Med Child Neuro

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“…The psychometric properties of any measure are not universal; rather, they must be specific to each issue/question and to the population being reported on. Of importance to pediatric neurology is the increasing interest in the role and capability of children and adolescents to report their own health and QoL 14 …”

Section: Essential Properties Of Measuresmentioning

confidence: 99%

“…Of importance to pediatric neurology is the increasing interest in the role and capability of children and adolescents to report their own health and QoL. 14…”

Section: Essential Properties Of Measures Promsmentioning

confidence: 99%

Patient perspectives in pediatric neurology: a critical shift in the paradigm of outcome measurement

Ronen

Rosenbaum

Streiner

2021

Develop Med Child Neuro

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This review explores children’s self‐reported outcome measurements in pediatric neurology. We examine the following questions: (1) What is meant by patient‐reported health, functioning, and quality of life outcomes? (2) How can patients express whether the interventions they receive do more good than harm? (3) Why and how should pediatric neurology patients help determine the outcomes of interest? (4) What tools and recommendations are available to evaluate the outcomes of interest? Applying patients’ perspectives across the processes of evaluation of medical interventions has become an important expectation. These developments, consistent with current healthcare goals, coincide with the evolution of pediatric neurology into a sophisticated diagnostic–interventional field that aims to prolong survival, decrease impairments and symptoms, and improve patients’ well‐being – the recognized essential endpoints of interest in all medicine.

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“…Health regulatory authorities recommend assessing QoL through a patient-centred approach as a part of medical product development in order to incorporate patient experience data and other relevant information from patients and caregivers [ 14 ]. In the absence of specific quantitative tools to obtain patient reported outcome measures in the context of dRTA, qualitative research can be useful to document patient reported outcomes [ 15 – 17 ]. Due to the lack of responsiveness of existing generic patient reported outcomes and the challenge in developing specific patient reported outcomes questionnaires in rare diseases, qualitative interviews seem to be the most adapted strategy to measure patient experiences in patients with dRTA.…”

Section: Introductionmentioning

confidence: 99%

Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study

Acquadro

Marrel

Manso-Silván

et al. 2022

Orphanet J Rare Dis

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Background Consequences of distal renal tubular acidosis (dRTA) on growth, bone and kidney, sometimes associated with hearing loss, may significantly affect quality of life (QoL). This descriptive qualitative study explores QoL linked to dRTA and gathers the impressions of patients with this rare disease (and caregivers) 5 years after enrolment in a clinical study, during which patients were treated with ADV7103, a prolonged-release granule formulation combining potassium citrate and potassium bicarbonate. Semi-structured, one-hour interviews with 6 adult and 13 paediatric patients with a confirmed diagnosis of dRTA and with parents of paediatric patients were performed using an interview guide. Qualitative analysis of anonymized interview transcripts based on grounded theory was conducted. Results The main QoL domains impacted by dRTA and its treatment were education/work, social/family life, and emotional and physical well-being. ADV7103 (administered twice daily) was compared with the standard of care (SoC) taken before study entry (more than twice daily). Patients/parents reported that switching from previous SoC to ADV7103 had changed their lives: Difficulties at school due to burdensome administrative issues and need to explain disease and treatment affecting all families of paediatric patients (n = 13) disappeared, facilitating parents who had stopped working (to deal with their child’s treatment) to return to work, Family functioning was improved (n = 18), as travel and holidays became easier to organise and patients/parents stopped thinking about managing treatment daily/nightly, reducing tension in the family or couple, The emotional burden of disease perceived was relieved (n = 12) in the absence of treatment-related invasive questions from others, Gastro-intestinal adverse events and taste problems improved with ADV7103 (n = 18) and better compliance led to milder physical impacts and less need to be hospitalised. The mean satisfaction score with ADV7103 compared to SoC was 9 out of 10 (10 = very satisfied). ADV7103 exceeded or met the expectations of 14 out of 17 patients that commented on that. Conclusions Qualitative interviews show that dRTA and its treatment have a significant impact on QoL of patients and parents and that ADV7103 helps improve daily-life and reduces treatment burden, resulting in greater overall satisfaction of the patients and their families. Trial registration EU Clinical Trials Register, EudraCT 2013-003828-36 on the 3rd of September 2013.

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Engaging the Voices of Children: A Scoping Review of How Children and Adolescents Are Involved in the Development of Quality-of-Life–Related Measures

Cited by 10 publications

References 130 publications

Childhood disability: The importance of context

Childhood disability: The importance of context

Patient perspectives in pediatric neurology: a critical shift in the paradigm of outcome measurement

Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study

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