Stratified medicine in paediatrics is increasingly becoming a reality, as our understanding of disease pathogenesis improves and novel treatment targets emerge. We have already seen some success in paediatrics in targeted therapies such as cystic fibrosis for specific cystic fibrosis transmembrane conductance regulator variants. With the increased speed and decreased cost of processing and analysing data from rare disease registries, we are increasingly able to use a systems biology approach (including ‘-omics’) to screen across populations for molecules and genes of interest. Improving our understanding of the molecular mechanisms underlying disease, and how to classify patients according to these will lead the way for targeted therapies for individual patients. This review article will summarise how ‘big data’ and the ‘omics’ are being used and developed, and taking examples from paediatric renal medicine and rheumatology, demonstrate progress being made towards stratified medicine for children.
- paediatric practice
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MAS and AVR contributed equally.
Contributors WYD wrote the first draft, revised the manuscript and approved the final version. AVR and MAS conceived the work, edited the manuscript and approved the final version. MWB edited the manuscript and approved the final version.
Funding WYD is funded by a Kidney Research UK Clinical Research Fellowship.
Competing interests MAS has external consultancy roles with UCB, Retrophin and Pfizer. AVR has received speaker fees/consultancy for AbbVie, Eli Lilly, UCB and SOBI.
Patient consent Not required.
Provenance and peer review Commissioned; externally peer reviewed.
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