Systematic review of instruments for the assessment of patient-reported outcomes and quality of life in patients with childhood glaucoma

Author: Stingl JV, Ortolano LC, Azuara-Blanco A, Hoffmann EM.

Geographical coverage: Not reported

Sector: Impact/Economic Evaluation

Sub-sector: Patient-Reported Outcomes and Quality of Life

Equity focus: Children or adolescents

Study population: Children or adolescents (age < 18 years) with glaucoma, and adults with childhood or juvenile onset of glaucoma.

Review type: Effectiveness review

Quantitative synthesis method: Meta-analysis

Qualitative synthesis method: Not applicable

Background

Glaucoma is the second leading cause of blindness worldwide, affecting about 1.4% to 2.0% of adults in Europe. Numerous patient-reported outcome measures (PROMs) exist for assessing vision-related and health-related quality of life in adults with glaucoma. However, none are specifically designed and validated for children. Childhood glaucoma is rare but serious, occurring in 10 to 30 per 100,000 births in Europe, often requiring multiple surgeries and lifelong treatment. Existing PROMs may not fully capture the impacts on children, such as developmental issues, caregiver burden, and difficulties with treatment adherence. For younger children, assessments are typically made by caregivers. Evaluating quality of life in children with glaucoma is vital, but the best patient‑reported outcome measure (PROM) for this purpose is unclear. This review aims to identify PROMs used for children and adolescents with glaucoma, assess their quality, and determine their suitability for evaluating quality of life in this group.

Objectives

To identify PROMs used in children and adolescents with glaucoma and assess their methodological quality.

Main findings

The review identified both vision-specific and generic health-related patient-reported outcome (PRO) instruments used in childhood glaucoma cohorts. However, no instrument specifically designed for childhood glaucoma was found. This gap may lead to the omission of critical disease-specific factors, such as lifelong eye drop use, repeated surgeries, and the heritability of glaucoma when assessing the quality of life (QoL) in affected children.

In total, 11 studies using 10 different PROMs were identified and the 10 PROMs were included in this review. All PROMs were developed and validated in English language. Of the 10 PROMs reviewed, six were vision-specific and four were generic health instruments. Four vision-specific and two generic health PROMs were designed for children/adolescents, while the remaining were for adults. The instruments assessed functional visual ability, vision-related quality of life (VRQoL), health-related quality of life (HRQoL), and life satisfaction. Seven PROMs received high-quality ratings (5/7), but none were specifically developed for childhood glaucoma or included patient perspectives during development.

Instruments varied in classification based on the World Health Organisation International Classification of Functioning, Disability and Health (WHO ICF) framework, with differences in their ability to capture disease-specific factors. Generic PROMs were less responsive to glaucoma severity, while vision-specific measures better reflected visual impairment and treatment impact. The review highlighted the need for a childhood glaucoma-specific PROM to improve assessment and patient care.

Methodology

The authors searched four databases on 4 November 2022, including MEDLINE (PubMed), PsycINFO (EBSCOhost), Cochrane Library, and Web of Science. They limited the search to publications from 2012 to 2022 due to advancements in healthcare and PROM development.

The review included peer-reviewed studies on PROs, such as QoL or functional visual ability (FVA) in childhood or juvenile-onset glaucoma. Articles needed to be in English, Spanish, or German, with at least 50% of participants under 18, later expanded to include adults with childhood glaucoma. Studies needed to develop, evaluate, or report on a PROM, answered by children or caregivers. Two reviewers independently screened the articles against the eligibility criteria, resolving disagreements through discussion or a third reviewer.

Quality assessment of the corresponding PRO development studies for the identified instruments was conducted using a published tool. Data extraction was done independently by two reviewers, resolving disagreements through discussion or a third reviewer. Baseline characteristics of PROMs, including construct, target population, administration mode, publication year, language, and translations, were compiled. Instruments were categorised as vision-specific or generic health and as child- or adult-targeted. Each item was classified using the WHO ICF framework into body structure/function (B), activity (A), participation (P), and satisfaction (S). Instruments were further grouped as impairment measures (B only), status measures (B, A, and P), disability measures (A and P only), or satisfaction measures (S only).

Applicability/external validity

The review did not address the applicability or external validity of its findings in detail but noted limitations affecting generalisability: language restrictions (English, German, Spanish) excluding other relevant studies, and a 10-year search window missing older, possibly more specific childhood glaucoma PROMs.

Geographic focus

Geographic location of included studies was not reported by the authors.

Summary of quality assessment

The review authors did not report checking reference lists of included studies or contacting authors/experts for additional data. Additionally, the review did not provide a list of excluded studies, and the findings were not reported by the risk of bias status.