Abstract
The purpose of this study is to describe the burden of health care utilisation and early education outcomes of children with and without rare diseases. Using the Born in Bradford birth cohort and its linked routine health care and education data, we looked at cumulative primary care episodes, hospital admissions and medication prescriptions. We assessed education outcomes using Early Years Foundation Stage Profile and the National Curriculum Tests-Key Stage 1 results. Among analytical sample of 13,858, 1711 or 12.3% children were identified with a rare disease by an average age of 14 years. Children with rare diseases were twice more likely to be admitted to hospital compared to children without. Average length of stay was around 5 days in those with rare diseases group compared to less than 1 day in those without. The average number of primary care episodes was 45.4 in children with rare diseases and 28.2 visits in those without. These children were over three times more likely to be on multiple medications. Children with rare diseases had 30% higher risk of being below academic expectations at Foundation Year and, depending on subject, between 50 and 60% higher risks at KS1 tests.
Conclusions: Children with rare diseases are significantly more likely to have increased primary care episodes and to have more regular medications. They are likely to have more hospital admissions with a longer stay also. Educationally, they are at higher risk of failing to achieve expected standards in early-year settings.
What is Known: • Existing studies of rare diseases have used cross sectional data to describe secondary care data. Previous research has not explored the impact of rare diseases on academic outcomes in children. | |
What is New: • Using Born in Bradford birth cohort and its linked primary and secondary care data, this study provides the most comprehensive estimate of prevalence of rare diseases in any study to date. Children with rare diseases were not only significantly more likely to have contact with primary care and to be admitted to hospitals; they were also more like to be on more regular medications and had higher risk of not achieving expected standards in early-year education outcomes. • Our study is unusual in being able to access linked health and education data and reinforces the importance of adopting a whole system approach to children’s health and wellbeing that recognises the close links between health and education. |
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Abbreviations
- BiB:
-
Born in Bradford
- BNF:
-
British National Formulary
- CI:
-
Confidence interval
- ELGs:
-
Early learning goals
- EYFS:
-
Early years foundation stage profile
- GLD:
-
Good level of development
- ICD 10:
-
International Classification of Diseases 10th Revision
- IQR:
-
Interquartile ranges
- RD:
-
Rare diseases
- RR:
-
Relative risk
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All authors conceptualised the study. RL and BH drafted a first version of the paper. BH and AH conducted the data analysis. RL, DM, SO and JW provided supervision during this process and critically reviewed the manuscript. All authors approved the final version of this manuscript.
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This research was approved by the Born in Bradford Ethics Committee, reference number 07/H1302/112 and conducted according to the Helsinki principles. Appropriate guardians of children in the Born in Bradford cohort gave their written informed consent for scientific use of their data.
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Written informed consent was obtained from all Born in Bradford cohort studies participants. This study conducted secondary analysis and no additional data collection was conducted.
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Lodh, R., Hou, B., Hough, A. et al. Health care utilisation and education outcomes of children with rare diseases: a born in Bradford cohort study. Eur J Pediatr 182, 5511–5517 (2023). https://doi.org/10.1007/s00431-023-05225-4
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DOI: https://doi.org/10.1007/s00431-023-05225-4