Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12621000657820
Ethics application status
Approved
Date submitted
2/04/2021
Date registered
31/05/2021
Date last updated
31/05/2021
Date data sharing statement initially provided
31/05/2021
Type of registration
Prospectively registered

Titles & IDs
Public title
Quality of Kids' Lives Study – Finding the best way to measure kids’ health
Scientific title
Paediatric Health-Related Quality of Life Measurement and Validation: An Australian Multi Instrument Comparison Study
Secondary ID [1] 303841 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Asthma 321376 0
Autism Spectrum Disorder 321377 0
Sleep problems 321378 0
Dental decay 321379 0
Anxiety 321380 0
Attention Deficit Hyperactivity Disorder 321381 0
Acutely unwell requiring Emergency medicine 321386 0
Behaviour problems 321387 0
Bowel problems 321388 0
Diabetes 321389 0
Neurological disorders 321390 0
Gynaecology problems 321391 0
Developmental disabilities 321392 0
Plastic surgery 321395 0
Colorectal surgery 321396 0
Dental problems 321397 0
Depression 321398 0
Developmental delay 321761 0
Bladder problems 321762 0
Maxillofacial problems 321763 0
Condition category
Condition code
Respiratory 319151 319151 0 0
Asthma
Mental Health 319152 319152 0 0
Autistic spectrum disorders
Oral and Gastrointestinal 319154 319154 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Mental Health 319155 319155 0 0
Anxiety
Mental Health 319156 319156 0 0
Depression
Mental Health 319157 319157 0 0
Other mental health disorders
Surgery 319160 319160 0 0
Other surgery
Neurological 319161 319161 0 0
Epilepsy
Emergency medicine 319162 319162 0 0
Other emergency care

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
This is a multi-instrument comparison study involving the prospective collection of several paediatric health related quality of life (HRQoL) instruments via two surveys, an initial 15-30-minute survey and a retest follow-up 5 minute survey 2-8 weeks later (or two days later if allocated to shorter follow-up group). The initial survey will include the following questionnaires: Informed Consent, Demographic Information, Carer Quality of Life (EQ-HWB), Strengths and Difficulties Questionnaire (SDQ), Pediatric Quality of Life Inventory (PedsQL), Toddler and Infant Health Related Quality of Life (TANDI), EQ-5D-Y (3L and 5L), EQ-5D-Y VAS, Child Health Utility (CHU9D), Adolescent Quality of Life (AQoL), Health Utilities Index (HUI2/3), EQ-5D-5L and PROMIS-25 (Pediatric Profile) and relevant disease-specific questionnaires: Asthma PedsQL, KIDSCREEN-27, Sleep disturbances Scale For Children (SDSC), Child Perceptions Questionnaire (CPQ), Revised Child Anxiety and Depression Scale 25 (RCADS-25), ADHD-Symptoms and Normal-Behaviors (SWAN). Participants will also be asked a self-reported difficulty question for each instrument. The follow-up survey will include the following questionnaires: PedsQL, TANDI, EQ-5D-Y (3L and 5L), EQ-5D-Y VAS, and CHU9D, as well as self-reported difficulty for each instrument. Caregivers (proxy) will always complete the demographic section and carer quality of life instrument. If the study child is under the age of 7 years the caregiver (proxy) will complete all questions and instruments of both surveys. If the study child is aged 7 years or above and the caregiver determines the child is able to answer questions about their own health and wellbeing, the child will be asked to provide consent and to answer (self-report) the pediatric quality of life instruments of both surveys.

This study will compare the performance of the aforementioned HRQoL instruments in terms of acceptability, feasibility, reliability, validity and sensitivity within the Australian context as well compare child age and disease group.

We plan to collect data on N=4,900 Australian children. There will be three key samples within the study cohort: (1) n=1,000 children from The Royal Children's Hospital, (2) n=2,400 children with specific disease conditions recruited via online survey panels (n=400 from each disease group: ADHD, anxiety or depression, ASD, asthma, dental decay and sleep problems) and (3) n=1,500 population sample recruited via online survey panels. A small subset of participants will be selected at random to complete observation sessions at a shorter follow-up timepoint at 2 days post initial survey (n=200).

Caregivers of children from The Royal Children's Hospital sample (n=1,000) will predominantly be recruited by research assistants who will approach families face-to-face in waiting areas across the hospital and ask caregivers to complete the initial survey on an iPad or their own device (will be provided a QR code or text a link on the spot). Some participants will be recruited via social media adverts or adverts on the virtual telehealth waiting room, these participants will complete the survey online via REDcap (with a link to the survey provided in the adverts). The follow-up survey will be delivered online.

Caregivers from the online panel samples (n=2,400 children with specific disease conditions and n=1,500 population sample) will be recruited via an external online survey panel company. Participants from these samples will complete both the initial and follow-up survey online.

Participants where the study child is aged 2-4 years may be enrolled into both the Quality of Little Lives Study and this study. The same specific disease subgroups will be assessed for this study.
Intervention code [1] 320146 0
Not applicable
Comparator / control treatment
The performance of instruments will be compared by child age and disease groups. Disease groups (Hospital sample, ADHD sample, anxiety or depression sample, ASD sample, asthma sample, dental decay sample and sleep problems sample) will be compared with the general population sample.
Control group
Active

Outcomes
Primary outcome [1] 327030 0
Validity (the degree to which the HRQoL instrument measures the construct(s) it purports to measure) of currently available pediatric HRQoL instruments (Global Health Measure, PedsQL, TANDI, EQ-5D-Y (3L and 5L), CHU9D, HUI2/3, AQoL, PRMOIS-25 and EQ-5D-5L). Validity of instruments will be measured using within-scale analysis (measured using factor analysis), known group differences (measured by descriptively comparing a priori assumptions regarding expected differences between disease groups and healthy children), convergent validity (measured by analysing the correlation of similar constructs from different instruments hypothesised to measure similar constructs) and discriminant validity (measured by analysing whether dimension responses are independent of child age).
Timepoint [1] 327030 0
At both initial and follow-up survey.
Primary outcome [2] 327031 0
Consistency (the degree to which the summary and dimension specific responses on each instrument are consistent with dimension and summary responses of other similar instruments) of currently available paediatric HRQoL instruments (Global Health Measure, PedsQL, TANDI, EQ-5D-Y (3L and 5L), CHU9D, HUI2/3, AQoL, PRMOIS-25 and EQ-5D-5L). Consistency of instruments will be assessed by comparing the consistency of summary and dimension specific responses on each instrument.
Timepoint [2] 327031 0
At both initial and follow-up survey.
Primary outcome [3] 327044 0
Acceptability and feasibility (composite primary outcome) of currently available paediatric HRQoL instruments (Global Health Measure, PedsQL, TANDI, EQ-5D-Y (3L and 5L), CHU9D, HUI2/3, AQoL, PRMOIS-25 and EQ-5D-5L). Acceptability and feasibility of instruments will be measured by assessing the time to complete instruments (measured using web analytics), completeness of data (measured by analysing data missingness) and reported difficulty (measured by survey question asking participants to report how difficult instrument was to complete, a single item question designed specifically for this study).
Timepoint [3] 327044 0
At both initial and follow-up survey.
Secondary outcome [1] 393517 0
Primary outcome: Reliability (the stability of the instrument) of currently available pediatric HRQoL instruments (Global Health Measure, PedsQL, TANDI, EQ-5D-Y (3L and 5L), CHU9D, HUI2/3, AQoL, PRMOIS-25 and EQ-5D-5L). Reliability of instruments will be assessed using test-retest reliability, measured by agreement on dimension-level responses between the initial survey to the re-test survey 2 days later.
Timepoint [1] 393517 0
At both initial and follow-up survey.
Secondary outcome [2] 393583 0
Primary outcome: Responsiveness (the ability of an instrument to detect change in the construct to be measured) of currently available pediatric HRQoL instruments (Global Health Measure, PedsQL, TANDI, EQ-5D-Y (3L and 5L), CHU9D, HUI2/3, AQoL, PRMOIS-25 and EQ-5D-5L). Responsiveness of instruments will be assessed using dimension level responses from children whose proxy respondents reported a change in general status from the initial survey to the follow-up survey 2-8 weeks later in comparison to those not showing a change. This will be assessed to determine the extent to which instruments are responsive to change in general health status.
Timepoint [2] 393583 0
Initial and follow-up survey.

Eligibility
Key inclusion criteria
Parent/caregiver of a child(ren) aged of 2-18 years old (inclusive) at enrolment. Additionally, children aged 7 years or older at enrolment whose caregiver/parent has identified they are currently capable of completing questions about their health are also eligible to complete part of the survey.
Minimum age
7 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
• Is unable to communicate in written English. Language will be simplified as much as possible to allow inclusion of caregivers who might have limited English proficiency
• Reside outside of Australia
• Unable to answer or comprehend questions

Study design
Purpose
Duration
Cross-sectional
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
The performance of the various paediatric QoL instruments will be analysed and compared at summary, dimension and item levels using psychometric analyses. Additionally, the feasibility, acceptability and responsiveness of the various instruments will be compared. We will also look at a subset of disease groups to see how generic HRQoL instruments perform compared with disease-specific instruments.

Due to the observational and methodological nature of this study design it is not possible to calculate a sample size a priori. The sample size of n=4,900 was estimated from a similar adult study and a review of paediatric QoL studies. We plan to recruit n=1,000 participants from The Royal Children's Hospital and n=3,900 participants via online survey panels. The sample size will allow for assessment of validity of the HRQoL instruments in a variety of childhood conditions both in and out of the hospital setting ranging from acute to chronic and across severities ranging from well children to those that are very sick. This diversity in the sample is important to determine the validity and applicability of the HRQoL instruments to groups of children.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
8/06/2021
Actual
Date of last participant enrolment
Anticipated
31/01/2022
Actual
Date of last data collection
Anticipated
14/02/2022
Actual
Sample size
Target
4900
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 19044 0
The Royal Childrens Hospital - Parkville
Recruitment postcode(s) [1] 33591 0
3052 - Parkville

Funding & Sponsors
Funding source category [1] 308235 0
Government body
Name [1] 308235 0
Department of Health
Country [1] 308235 0
Australia
Primary sponsor type
Other
Name
Murdoch Children's Research Institute
Address
50 Flemington Road, Parkville, Victoria, 3052
Country
Australia
Secondary sponsor category [1] 309035 0
None
Name [1] 309035 0
Address [1] 309035 0
Country [1] 309035 0
Other collaborator category [1] 281710 0
University
Name [1] 281710 0
Health Economics Unit, The University of Melbourne
Address [1] 281710 0
Level 4, 207 Bouverie Street
The University of Melbourne
Melbourne School of Population and Global Health
Parkville, Victoria, 3010, Australia
Country [1] 281710 0
Australia
Other collaborator category [2] 281711 0
University
Name [2] 281711 0
Centre for Health Economics Research and Evaluation, The University Technology Sydney
Address [2] 281711 0
Level 2, Building 5, Block D
1-59 Quay St, Haymarket NSW 2000
Country [2] 281711 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 308217 0
The Royal Children's Hospital Human Research Ethics Committee
Ethics committee address [1] 308217 0
Research Ethics & Governance
The Royal Children's Hospital
Level 4, South Building
50 Flemington Road
Parkville Vic 3052
Ethics committee country [1] 308217 0
Australia
Date submitted for ethics approval [1] 308217 0
25/02/2021
Approval date [1] 308217 0
20/05/2021
Ethics approval number [1] 308217 0
HREC/71872/RCHM-2021

Summary
Brief summary
Each year, researchers and doctors work to find better tests, treatments, and services for children. They often use questionnaires that ask about a child’s general health (also called ‘health related quality of life’) to understand how these tests, treatments and services improve children’s lives. However, sometimes this can be hard as there is a lack of sound evidence on how well these health related quality of life (HRQoL) questionnaires perform in children. More evidence on the performance of these questionnaires is required in Australia to guide decision making. We hope the results of this work may help us to better measure overall health in children in order to provide better health care to children in the future.

This study will compare lots of different HRQoL questionnaires in children (sometimes called a ‘multi-instrument comparison study’). The study will involve an initial and follow-up survey. The follow-up survey will be a simplified version of the initial survey that will be sent out 2-8 weeks after completion of the first survey. For the small sub-set (N=200) of participants the follow-up survey will be sent two days after the completion of the initial survey. The performance of these various child HRQoL questionnaires will be analysed and compared. We will also look at a subset of disease groups to see how generic HRQoL questionnaires perform compared with disease-specific questionnaires. We plan collect data on 4,900 Australian children (from children who are well through to those that are very sick).

The objectives of the study are to: (1) understand how well various child HRQoL questionnaires can be filled out by parents and how well they measure problems and improvements in health (i.e. consistency, acceptability, feasibility, reliability, responsiveness and validity) and (2) provide government, health practitioners and researchers with a practical set of tools that are ‘fit for purpose’ in judging the effectiveness and cost effectiveness of child health interventions.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 109966 0
A/Prof Kim Dalziel
Address 109966 0
Health Economics Unit
Level 4, 207 Bouverie Street
The University of Melbourne
Melbourne School of Population and Global Health
Parkville, Victoria, 3010, Australia
Country 109966 0
Australia
Phone 109966 0
+61 3 9345 5522
Fax 109966 0
Email 109966 0
[email protected]
Contact person for public queries
Name 109967 0
A/Prof Kim Dalziel
Address 109967 0
Health Economics Unit
Level 4, 207 Bouverie Street
The University of Melbourne
Melbourne School of Population and Global Health
Parkville, Victoria, 3010, Australia
Country 109967 0
Australia
Phone 109967 0
+61 3 9345 5522
Fax 109967 0
Email 109967 0
[email protected]
Contact person for scientific queries
Name 109968 0
A/Prof Kim Dalziel
Address 109968 0
Health Economics Unit
Level 4, 207 Bouverie Street
The University of Melbourne
Melbourne School of Population and Global Health
Parkville, Victoria, 3010, Australia
Country 109968 0
Australia
Phone 109968 0
+61 3 9345 5522
Fax 109968 0
Email 109968 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Due to privacy restrictions of health data and the potentially sensitive and identifiable nature of this data no IPD will be publicly available.


What supporting documents are/will be available?

Current supporting documents:
Doc. No.TypeCitationLinkEmailOther DetailsAttachment
11220Study protocol    381727-(Uploaded-30-04-2021-15-08-59)-Study-related document.docx
11221Informed consent form    Hospital informed consent form. 381727-(Uploaded-30-04-2021-15-12-11)-Study-related document.docx
11222Informed consent form    Online panel informed consent form.


Updated to:
Doc. No.TypeCitationLinkEmailOther DetailsAttachment
11220Study protocol    381727-(Uploaded-16-02-2022-10-13-53)-Study-related document.docx
11221Informed consent form    Hospital informed consent form. 381727-(Uploaded-30-04-2021-15-12-11)-Study-related document.docx
11222Informed consent form    Online panel informed consent form.

Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseComparing the Psychometric Performance of Generic Paediatric Health-Related Quality of Life Instruments in Children and Adolescents with ADHD, Anxiety and/or Depression.2024https://dx.doi.org/10.1007/s40273-024-01354-2
N.B. These documents automatically identified may not have been verified by the study sponsor.