Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases (SeeMyLife)

Rare Eye Diseases (RED) are the leading cause of severe visual impairment/ blindness (SVI/B) in children in Europe. This sensory disability with its accompanying psychological distress hugely impacts their lives and their families. Understanding this impact, at a patient centred level, is key in care, in shared decision making, in developing therapies, and in improving social integration and participation about the standard rules of the United Nations (UN) and the European Union (EU) (prevention, non-discrimination, equal opportunities, accessibility, etc.). However, current tools to evaluate vision related (VR) quality of life (QoL) VR-QoL disregard age and cultural differences. There is a lack knowledge on how the disease matters at child's level. Instruments capable of yielding high-quality data, psychometrically robust and comply with regulatory requirements remain to be developed.

To fill this gap, SeeMyLife will use multilevel concurrent mixed method research combining quantitative studies and qualitative studies. The quantitative approach is based on (i) cross culturally translated validated VR-QoL questionnaires for children and teenagers (Functional Vision Questionnaire for Children and Young People - FVQ-CYP and Vision-related Quality of Life Questionnaire for Children and Young People - VQoL-CYP) and (ii) on caregiver's questionnaires addressing participation and environment (Participation and Environment Measure - Children and Youth - PEM-CY). To fully capture the picture of the child/teenager personal life the investigators will reinforce their investigations by in depth qualitative socio-anthropologic study with semi directive field interviews and fieldwork (to observe closely the living conditions of the children) to address how their impairment affects their wellbeing, social integration, and how they feel about medical and social interventions. Data analysis will use an integrated mixed method strategy to validate the quantitative tools and deliver a holistic QoL transnational tool.

The SeeMyLife project will provide (i) robust patient self-reported tools that will be then used in care and research (especially with the rise in novel therapies) as a standard as well as (ii) highly awaited knowledge about the SVI/B patient's position within his own life course, within his family and in relation to health and social care actors.

Study Overview

• Status: Recruiting
• Conditions: Quality of Life; Eye Diseases; Blindness; Severe Loss of Vision
• Intervention / Treatment: Other: Mixed Methods Research (MMR) using quantitative (QUAN) and qualitative (QUAL) tools

• Study Type: Observational
• Enrollment (Estimated): 154

Contacts and Locations

Study Locations

• France
  • Strasbourg, France, 67091
    • Recruiting
    • Centre de Référence pour les Affections Rares en Génétique Ophtalmologique (CARGO), Hôpitaux Universitaires
    • Principal Investigator: Hélène DOLLFUS, MD
    • Contact: Hélène DOLLFUS, MD; Phone Number: 33.3.88.12.81.20; Email: helene.dollfus@chru-strasbourg.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Children (age 8-12) and teenagers (age 13-18) with various levels of visual impairment defined according to World Health Organization (WHO) International Classification of Diseases (ICD) 10 - WHO ICD 10 [best-corrected visual acuity ≤ 0.3 decimal or ≤ 6/18], and their parents/caregivers.

Description

Inclusion Criteria:

• Children (age 8-12) and teenagers (age 13-18) with various levels of visual impairment defined according to World Health Organization (WHO) International Classification of Diseases (ICD) 10 - WHO ICD 10 [best-corrected visual acuity ≤ 0.3 decimal or ≤ 6/18], and their parents/caregivers.

Exclusion Criteria:

• Children, teenagers, and caregivers:

  1. who lack the ability to respond in a reliable way to the questions on how they feel about their visual impairment (patients with mild intellectual or cognitive deficiency may be able to reply accurately as opposed to cases with severe intellectual disability)
  2. with functional or non-ophthalmic reasons of visual impairment
  3. unable to provide consent/assent;
  4. who do not speak/read the language

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

children and teenagers affected by Rare Eye Diseases; A Mixed Methods Research (MMR) using quantitative (QUAN) Vision Related-QoL (VR-QoL = FVQ-CYP and VQoL-CYP) tools and psychosocial PEM-CY, and qualitative (QUAL) socio-anthropologic investigations can capture reliably children's and teenagers' Patient-Reported Outcome (PRO).

Other: Mixed Methods Research (MMR) using quantitative (QUAN) and qualitative (QUAL) tools; A Mixed Methods Research (MMR) using quantitative (QUAN) Vision Related-QoL (VR-QoL = FVQ-CYP and VQoL-CYP) tools and psychosocial PEM-CY, and qualitative (QUAL) socio-anthropologic investigations can capture reliably children's and teenagers' Patient-Reported Outcome (PRO). The QUAN study is a cross-sectional study including short-term re-test repetition (children/teenagers only) using the following PROMs for children/teenagers: Self-reported questionnaires for patients (FVQ-CYP and VQoL-CYP); For caregivers: PEM-CY questionnaires The QUAL study will rely on the following instruments: Semi-directive interviews,; Focus groups,; Field observation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
real-life impact of SVI/B in children and teenagers affected by Rare Eye Diseases	To elucidate whether Patient-Reported Outcome Measures (PROMs) reflect how children/teenagers perceive their lives to be impacted by Rara Eye Diseases using Vision-related Quality of Life Questionnaire for Children and Young People - VQoL-CYP	12 months

Collaborators and Investigators

• Sponsor: University Hospital, Strasbourg, France

Study record dates

Study Major Dates

• Study Start (Actual): July 17, 2024
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026

Study Registration Dates

• First Submitted: September 26, 2023
• First Submitted That Met QC Criteria: October 3, 2023
• First Posted (Actual): October 6, 2023

Study Record Updates

• Last Update Posted (Actual): August 29, 2024
• Last Update Submitted That Met QC Criteria: August 27, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Sensation Disorders; Vision Disorders; Eye Diseases; Blindness
• Other Study ID Numbers: 8826

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No