Evaluating the Quantity and Quality of Health Economic Literature in Blinding Childhood Disorders: A Systematic Literature Review.

BACKGROUND: Evidence on the socioeconomic burden associated with childhood visual impairment, severe visual impairment and blindness (VI/SVI/BL) is needed to inform economic evaluations of existing and emerging interventions aimed at protecting or improving vision. This study aimed to evaluate the quantity and quality of literature on resource use and/or costs associated with childhood VI/SVI/BL disorders. METHODS: PubMed, Web of Science (Ovid), the National Health Service (NHS) Economic Evaluation Database and grey literature were searched in November 2020. The PubMed search was rerun in February 2022. Original articles reporting unique estimates of resource use or cost data on conditions resulting in bilateral VI/SVI/BL were eligible for data extraction. Quality assessment (QA) was undertaken using the Drummond checklist adapted for cost-of-illness (COI) studies. RESULTS: We identified 31 eligible articles, 27 from the peer-reviewed literature and four from the grey literature. Two reported on resource use, and 29 reported on costs. Cerebral visual impairment and optic nerve disorders were not examined in any included studies, whereas retinopathy of prematurity was the most frequently examined condition. The quality of studies varied, with economic evaluations having higher mean QA scores (82%) compared to COI studies (77%). Deficiencies in reporting were seen, particularly in the clinical definitions of conditions in economic evaluations and a lack of discounting and sensitivity analyses in COI studies. CONCLUSIONS: There is sparse literature on resource use or costs associated with childhood visual impairment disorders. The first step in addressing this important evidence gap is to ensure core visual impairment outcomes are measured in future randomised control trials of interventions as well as cohort studies and are reported as a discrete health outcome.

Temporal trends in the epidemiology of childhood severe visual impairment and blindness in the UK.

BACKGROUND/AIMS: Understanding temporal trends in childhood visual disability is necessary for planning and evaluating clinical services and health policies. We investigate the changing epidemiology of severe visual impairment (SVI) and blindness (BL) in children in the UK in the 21st century. METHODS: Comparative analysis of two national population-based epidemiological studies of incident childhood SVI/BL (ICD-10 definition; visual acuity worse than 1.0 LogMAR in the better eye). We carry out comparative analysis of studies conducted in 2000 and 2015 using identical methods. RESULTS: Overall annual and cumulative incidence rates remained broadly stable in 2015 at 0.38 per 10 000 (95% CI 0.34 to 0.41) for 0-15 years old and 5.65 per 10 000 (5.16 to 6.18) by 16 years, respectively, and with annual incidence in infancy (3.52 per 10 000, 3.13 to 3.97) remaining considerably higher than any other age. Mortality among children diagnosed in infancy declined (from 61.4 to 25.6 per 1000), despite an increase (from 77% to 84%, p=0.037) in the overall proportion with significant non-ophthalmic impairments/disorders. The relative contribution of all the main groups of disorders increased over time, most notably cerebral visual impairment (from 50% to 61%). Aetiological factors operating prenatally continued to predominate, with an increased relative contribution of hereditary conditions in all children (from 35% to 57%, p<0.001). The substantially elevated rates for any ethnic minority group and those born preterm were unchanged, with amplification of increased rates associated with low birth weight. CONCLUSION: The changing landscape of healthcare and increased survival of affected children, is reflected in increasing clinical complexity and heterogeneity of all-cause SVI/BL alongside declining mortality.

Avoidable childhood blindness in a high-income country: findings from the British Childhood Visual Impairment and Blindness Study 2.

BACKGROUND/AIMS: Addressing childhood visual disability is an international priority, with data on causes needed to plan, implement and evaluate public health and clinical care. We have examined the contribution of 'avoidable' blinding disorders to childhood visual impairment, severe visual impairment and blindness (VI/SVIBL) in the UK. METHODS: National prospective observational longitudinal study, the British Childhood Visual Impairment and Blindness Study 2 (BCVIS2), of children (aged 18 years or under) newly diagnosed with vision worse than 0.48 logMAR (logarithm of the minimum angle of resolution) or equivalent in both eyes. Proportions of children with an 'avoidable' disorder comprising either a potentially preventable (isolated disorders with an effective intervention which reduces disease incidence) or treatable (isolated eye or vision disorders for which there are routinely available effective interventions able to improve vision or halt progressive visual loss) are reported. RESULTS: Of the 784 children within BCVIS2, isolated potentially preventable disorders were present in only 17% (132/784) and treatable disorders in an additional 13% (99/784). The most common treatable causes were cataract, retinopathy of prematurity and glaucoma. Of the 132 children with potentially preventable disease, 64 had hypoxic-ischaemic encephalopathy. Non-accidental injury accounted for almost two-thirds (11/16, 69%) of those with VI/SVIBL due to injury. CONCLUSION: Despite significant progress in the past decades in high-income countries, there remains a need to be vigilant about implementing preventive strategies and treatments. Attention to disorders that are currently neither preventable nor treatable remains a priority in these settings and will become increasingly important in lower-income and middle-income countries undergoing economic transition.

The role of screening and surveillance in the detection of childhood vision impairment and blindness in the UK.

OBJECTIVE: Understanding pathways to detection for childhood visual impairment (VI) is critical for planning services. We aimed to describe patterns of detection for childhood VI. DESIGN AND SETTING: Cross-sectional study using data from British Childhood Visual Impairment and Blindness Study 2. PATIENTS: Children newly diagnosed with VI, severe vision impairment or blindness (SVI/BL)-that is, visual acuity worse than logMAR 0.5 in both eyes-were identified through active surveillance, with data collection at diagnosis and 1 year later. OUTCOME MEASURE: Method of detection of vision/eyes problem. RESULTS: 784 children (45%, 356 girls) were identified, of whom 313 (40%) had VI, 471 (60%) had SVI/BL. Additional non-ophthalmic disorders or impairments (VI/SVI/BL 'plus'), were diagnosed in 72% (559/784). Of the 784, 173 children were detected through routine screening (22%), 248 through targeted examinations (32%) and 280 through family self-referral (36%). Parents and carers had only reported symptoms in 55% of children who manifested them, with evidence that families living in socioeconomically deprived areas were less likely to report concerns. Paediatricians were the professionals most likely to raise initial suspicion of visual disability. CONCLUSIONS: Our findings show that targeted screening and surveillance is important for the detection of full spectrum childhood visual impairment (VI/SVI/BL), as a significant proportion of children will not have symptoms, or their parents or carers will not report symptoms. As paediatricians were the professionals most commonly involved in detection, it would be helpful if their core competencies included the skills needed to undertake simple assessments of vision.

Visual impairment, severe visual impairment, and blindness in children in Britain (BCVIS2): a national observational study.

BACKGROUND: The WHO VISION 2020 global initiative against blindness, launched in 2000, prioritised childhood visual disability by aiming to end avoidable childhood blindness by 2020. However, progress has been hampered by the global paucity of epidemiological data concerning childhood visual disability. The British Childhood Visual Impairment and Blindness Study 2 (BCVIS2) was done to address this evidence gap. METHODS: BCVIS2 was a prospective UK-wide, cross-sectional, observational study to establish an inception cohort of children newly diagnosed with visual impairment. Opthalmologists and paediatricians reported cases from 89 hospitals and community centres across the UK. We included children aged 18 years or younger who were newly diagnosed with any condition causing impaired visual acuity to a level of 0·5 logMAR or worse (worse than 6/18 Snellen) in each eye, or equivalent vision as assessed by standard qualitative measures, between Oct 1, 2015, and Nov 1, 2016. Eligible children were notified simultaneously but independently by their managing ophthalmologists and paediatricians via the two national active surveillance schemes, the British Ophthalmological Surveillance Unit and the British Paediatric Surveillance Unit. Standardised detailed demographic, socioeconomic, and clinical data about detection, management, and treatment were collected at diagnosis and 1 year later. We calculated incidence estimates and relative rates by key sociodemographic factors. We did descriptive analyses of underlying ophthalmic disorders and non-ophthalmic comorbidities. FINDINGS: 61 (7%) of 845 eligible children initially notified were ineligible at follow-up because of improved vision after treatment. Thus, the study sample comprised 784 children with permanent newly-diagnosed all-cause visual impairment, severe visual impairment, or blindness. 559 (72%) of 778 children had clinically significant non-ophthalmic impairments or conditions. 28 (4%) of 784 children died within a year after diagnosis of visual disability (all had underlying systemic disorders). Incidence of visual disability in the first year of life was 5·19 per 10 000 children (95% CI 4·71-5·72), almost ten times higher than among 1-to-4-year-olds and between 20 times and 100 times higher than in the older age groups. The overall cumulative incidence (or lifetime risk) of visual impairment, severe visual impairment, or blindness was 10·03 per 10 000 children (9·35-10·76). Incidence rates were higher for those from any ethnic minority group, the lowest quintile of socioeconomic status, and those born preterm or with low birthweight. 345 (44%) of 784 children had a single affected anatomical site. Disorders of the brain and visual pathways affected 378 (48%) of 784 children. INTERPRETATION: BCVIS2 provides a contemporary snapshot of the heterogeneity, multi-morbidity, and vulnerability associated with childhood visual disability in a high-income country. These findings could facilitate developing and delivering health care and planning of interventional research. Our findings highlight the importance of including childhood visual disability as a sentinel event and metric in global child health initiatives. FUNDING: Fight for Sight, National Institute for Health Research, and Ulverscroft Foundation.

Protocol for a scoping review to map evidence from randomised controlled trials on paediatric eye disease to disease burden.

BACKGROUND: Currently, about 2 per 1000 children in the industrialised world are severely visually impaired or blind (SVI/BL) due to diverse uncommon conditions that are usually present from early infancy. The impact of SVI/BL is lifelong and life-changing. Thus, children are a priority in the WHO-led global initiative against avoidable blindness. The aim of this scoping review is to assess the current evidence base on interventions to prevent or treat the major causes of childhood SVI/BL, specifically the degree of alignment between robust interventional research (RCTs) and the burden (relative frequency) of the key causative disorders, identifying gaps in the evidence base for tackling childhood blindness. METHODS/DESIGN: We will perform a scoping review of the published literature of randomised controlled trials (RCTs) for clinical interventions that prevent or treat eye and vision diseases in children (<18 years old). Major electronic databases MEDLINE (PUBMED), EMBASE and the Cochrane CENTRAL will be searched to identify published trials using a comprehensive paediatric specific strategy informed by previous searches. The outcome of our study, randomised clinical trial activity, will be measured by the total number of RCTs and total paediatric participants randomised. The quantity and distribution of activity across diseases will be classified in the broad categories of anatomical site affected (per WHO taxonomy). The degree of alignment between paediatric trial activity and burden of SVI/BL disease (relative proportion) will be measured using a test of association (Spearman's correlation coefficient). DISCUSSION: Despite the global public health importance of childhood blindness, there has been no assessment of the completeness of the evidence base regarding clinical interventions to prevent or treat the causative disorders. This scoping review will measure the degree of alignment between the published evidence and the burden of disease to identify gaps in current knowledge and consider the underlying reasons, informing clinicians, policy makers and funders about research priorities.

Epidemiology of blindness in children.

An estimated 1.4 million of the world's children are blind. A blind child is more likely to live in socioeconomic deprivation, to be more frequently hospitalised during childhood and to die in childhood than a child not living with blindness. This update of a previous review on childhood visual impairment focuses on emerging therapies for children with severe visual disability (severe visual impairment and blindness or SVI/BL).For children in higher income countries, cerebral visual impairment and optic nerve anomalies remain the most common causes of SVI/BL, while retinopathy of prematurity (ROP) and cataract are now the most common avoidable causes. The constellation of causes of childhood blindness in lower income settings is shifting from infective and nutritional corneal opacities and congenital anomalies to more resemble the patterns seen in higher income settings. Improvements in maternal and neonatal health and investment in and maintenance of national ophthalmic care infrastructure are the key to reducing the burden of avoidable blindness. New therapeutic targets are emerging for childhood visual disorders, although the safety and efficacy of novel therapies for diseases such as ROP or retinal dystrophies are not yet clear. Population-based epidemiological research, particularly on cerebral visual impairment and optic nerve hypoplasia, is needed in order to improve understanding of risk factors and to inform and support the development of novel therapies for disorders currently considered 'untreatable'.

Emergency department based intervention with adolescent substance users: 10year economic and health outcomes.

BACKGROUND: Alcohol and other drug (AOD) use are significant cause of disease burden and costs among adolescents. METHODS: We conducted a randomized trial in hospital emergency departments (ED) following an AOD-related presentation, comparing usual care with brief advice and referral to link adolescents aged 12-19 years with external AOD services. Subsequently, we used health data linkage to assemble data on mortality, hospital admissions, ED attendances, out-patient mental health and use of opiate pharmacotherapies in the next 10 years. From these, treatment costs and rates of events were estimated and compared using generalized linear models. RESULTS: Those who received the intervention had lower costs ($22 versus $227: z=3.16, p=0.002) and rates (0.03 versus 0.25: z=2.57, p=0.010) of ED mental health AOD presentations. However, the intervention did not significantly reduce overall mean health costs per patient (intervention $58746 versus control $64833, p=0.800). Similarly, there was no significant difference in the costs associated with hospitalizations ($48920 versus $50911 p=0.924), overall ED presentations ($4266 versus $4150, p=0.916), out-patient mental health services ($4494 versus $7717, p=0.282), or opiate pharmacotherapies ($1013 versus $2054, p=0.209). Injecting drug use was a significant baseline predictor of subsequent costs in the cohort (z=2.64, p=0.008). CONCLUSIONS: An ED delivered intervention may reduce direct ED costs and subsequent ED AOD attendances. There was also some indication that overall costs may be impacted, with economically large but non-significant differences between the groups. The high costs and morbidity incurred by some of this cohort illustrate the importance of targeting high-risk adolescents.

Health care utilization and costs for children and adults with duchenne muscular dystrophy.

INTRODUCTION: Duchenne muscular dystrophy (DMD) is an incurable neuromuscular disorder of childhood. Healthcare, caregiving, and other resource needs of affected individuals are thought to be substantial; however, the economic burden associated with DMD has not yet been assessed specifically in Australia. METHODS: Australian households with a child with DMD were asked to complete a cross-sectional survey. Data were collected on annual resource utilization including hospital and medical services, equipment, home modifications, informal care, and working days lost. RESULTS: Mean healthcare costs were found to be $10,046 Australian dollars per affected individual and were markedly higher than average Australian health expenditures at each age group. The mean total cost was $46,700 (median $32,300), with healthcare costs contributing 22% of total costs. CONCLUSIONS: The annual economic cost of DMD was found to be high, reflecting a significant socioeconomic burden, especially in boys who reach adulthood, where household resource use and caregiving burden is highest. Muscle Nerve 53: 877-884, 2016.