Validation of the EQ-5D-Y-3L and EQ-5D-Y-5L for Paediatric Patients in China

NCT06873672 | Not Yet Recruiting DMC

• 2 other identifiers: 2024-IRB-1591721-RA
• Study Type: observational
• Target: 360
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational study is to validate the EQ-5D-Y-3L and EQ-5D-Y-5L in measuring health-related quality of life (HRQoL) for paediatric patients with different health conditions in China. The main research questions focus on comparing the psychometric performance of EQ-5D-Y-3L and EQ-5D-Y-5L, evaluating the differences between self-complete (SC), interviewer-administered (IA), and proxy-administered (Proxy) versions, and assessing their validity against other HRQoL measures like PedsQL. The study will recruit 360 inpatient-caregiver dyads from three disease groups, i.e., pneumonia, paediatric central nervous system (CNS) solid tumours, and immune thrombocytopenic purpura (ITP), using cross-sectional and longitudinal surveys. Baseline assessments will be conducted at hospital admission, with follow-up at hospital discharge (within 14 days). A qualitative component will explore acceptability and interpretation of EQ-5D-Y. The findings will contribute to the methodological development of EQ-5D-Y, with potential implications for HRQoL measurement and economic evaluations in paediatric healthcare.

Conditions

Quality of Life (QOL); Patient-Reported Outcome Measures (PROMs); Pneumonia Childhood; Neuroblastoma (NB); Wilms Tumour; Immune Thrombocytopenic Purpura ( ITP )

Keywords

Quality of life; children; psychometric measurements; Paediatric pneumonia; Paediatric central nervous system solid tumour; paediatric immune thrombocytopenic purpura; spillover effect

Outcome Measures

Primary Outcomes (1)

• Change in EQ-5D-Y Summary Scores across three disease groups from Baseline to Follow-up

  The EQ-5D-Y Summary Scores include: (1) Index Score which is derived using the Chinese value set for EQ-5D-Y-3L; (2) the Level Sum Score (LSS) which is calculated by summing the reported levels across five dimensions of the EQ-5D-Y-3L and EQ-5D-Y-5L (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), with higher scores indicating worse health; and (3) the visual analogue scale (EQ VAS), a self-reported global health assessment measured on a 0 to 100 scale, where 0 represents the worst imaginable health and 100 represents the best imaginable health. The change in these scores from baseline to follow-up will be analyzed to assess test-retest reliability, responsiveness and known-groups validity, and will be compared across the three administration modes (self-complete, interviewer-administered, and proxy-reported) and across three disease groups.

  From Day 1 (hospital admission) to hospital discharge (within 14 days)

Secondary Outcomes (1)

• Caregiver Burden and Spillover Effects Using EQ-5D-5L and EQ-HWB-S

  At hospital admission (Day 1)

Study Arms (4)

Children with pneumonia

Children with pneumonia will be recruited from inpatient wards at Guizhou Provincial People's Hospital and affiliated hospitals of Guizhou Medical University. These children are diagnosed with community-acquired pneumonia (CAP) and receive routine paediatric pneumonia treatment, including antibiotic therapy, oxygen support, intravenous fluids, and symptom monitoring, based on national clinical guidelines.

Children with central nervous system (CNS) solid tumours

Children with central nervous system (CNS) solid tumours will be recruited from The Children's Hospital affiliated with Zhejiang University School of Medicine. These children are diagnosed with CNS solid tumours, i.e., Neuroblastoma or Wilms tumour, and are either undergoing active treatment (including surgical resection, chemotherapy, and radiotherapy) or in post-treatment follow-up.

Children with immune thrombocytopenic purpura

Children with immune thrombocytopenic purpura (ITP) will be recruited from paediatric haematology departments at the No.1 Hospital Affiliated to Guizhou Medical University. These children are diagnosed with ITP, a rare autoimmune disorder characterized by low platelet counts and increased bleeding risk. Their treatment varies by disease severity, including observation, corticosteroids, intravenous immunoglobulin (IVIG), or thrombopoietin receptor agonists (TPO-RAs).

Family caregivers

Caregivers in this study include parents and family caregivers who provide long-term care and support for the child's well-being. Parents (biological or adoptive mothers and fathers) are the primary caregivers, responsible for medical decisions, daily care, and emotional support. Family caregivers, including grandparents, older siblings, aunts, or uncles, may assume caregiving roles due to parental absence or additional support needs, assisting with daily care, medical follow-ups, and emotional well-being.

Eligibility Criteria

• Age: 5 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Participants will be recruited from three tertiary hospitals different cities in China. The study will enroll paediatric patients aged 5-16 years who are receiving routine care for pneumonia, central nervous system (CNS) solid tumours, or immune thrombocytopenic purpura (ITP). These conditions represent common and clinically significant paediatric health issues requiring hospitalization or specialized treatment. The study will also recruit primary caregivers, who are typically parents or long-term family caregivers responsible for the child's daily care and medical decision-making.

You may qualify if:

• Children (Patients)
• Age: 5-16 years old.
• Diagnosis: Clinically confirmed diagnosis of one of the following conditions:
• Pneumonia (hospitalized due to community-acquired pneumonia); Central Nervous System (CNS) Solid Tumours (receiving active treatment or in post-treatment follow-up); Immune Thrombocytopenic Purpura (ITP) (diagnosed with acute or chronic ITP).
• Health Status: Cognitively and physically capable of completing HRQoL assessments (for self-complete versions).
• Language Proficiency: Able to understand and communicate in Mandarin Chinese.
• Consent: Written informed consent obtained from the primary caregiver and verbal or written assent from the child (if age-appropriate).
• Caregivers (Parents/Family Caregivers)
• Primary Caregiver: Must be a parent or a long-term family caregiver (e.g., grandparent, older sibling, aunt, or uncle).
• Involvement: Directly responsible for the daily care and medical decision-making of the child.
• Language Proficiency: Able to understand and complete both the self-complete and proxy-reported HRQoL assessments in Mandarin Chinese.
• Consent: Willing to provide informed consent for participation.

You may not qualify if:

• Children (Patients)
• Severe Cognitive or Developmental Impairment: Diagnosed with intellectual disability, autism spectrum disorder, or any neurological condition that prevents understanding and completing HRQoL assessments.
• Severe Physical Disability:
• Conditions that make it impossible to respond to surveys (e.g., advanced neuromuscular disease, severe paralysis).
• Comorbidities: Presence of another major chronic illness that could significantly affect HRQoL measurement (e.g., severe congenital heart disease, uncontrolled epilepsy).
• Recent Participation in Similar Studies:
• If the child has participated in another HRQoL validation study within the past 6 months.
• Inability to Complete Study Follow-Up: Expected to be transferred to another hospital or relocated during follow-up.
• Caregivers (Parents/Family Caregivers)
• Not the Primary Caregiver:
• Individuals who do not have daily caregiving responsibilities for the child.
• Cognitive Impairment or Severe Health Conditions:
• Caregivers with severe mental illness, dementia, or a medical condition that affects their ability to complete surveys.
• Language Barrier: Unable to understand and complete HRQoL assessments in Mandarin Chinese.
• Refusal to Participate: Caregivers who decline to provide informed consent.

Sponsors & Collaborators

• RenJi Hospital: lead
• The EuroQol Research Foundation: collaborator
• Guizhou Provincial People's Hospital: collaborator
• Guizhou Medical University: collaborator
• The Children's Hospital of Zhejiang University School of Medicine: collaborator
• The Affiliated Hospital Of Guizhou Medical University: collaborator

Study Sites (1)

Guizhou Medical University

Guiyang, Guizhou, 550025, China

Location

Related Publications (1)

• Qi X, Zhou W, Yang Z, Guannan B, Yang X, Zhang J, Busschbach J. Validation of EuroQol instruments in paediatric patients and their caregivers in China: protocol for a prospective observational study. BMJ Open. 2025 Jun 18;15(6):e102509. doi: 10.1136/bmjopen-2025-102509.

  PMID: 40533211 DERIVED

MeSH Terms

Conditions

Neuroblastoma; Wilms Tumor; Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Neoplasms, Complex and Mixed; Kidney Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplastic Syndromes, Hereditary; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Central Study Contacts

Wenjing Zhou, PhD

CONTACT

+31685281032; zhoujing2188@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Weeks
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 4, 2025
• First Posted: March 13, 2025
• Study Start: April 1, 2025
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: March 13, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL

Locations

CN (1)