Improving outcomes in paediatric surgery through the development and implementation of core outcome sets – the NETS studies
Keywords: Cohort studies
Evidence-based medicine
Paediatric Surgery
Epidemiology
Hirschsprung's disease
Gastroschisis
Core outcome sets

Background
Gold-standard medical practice involves utilising high quality evidence to support clinicians, patients, and their families in identifying the optimal treatment pathway for a particular individual. Currently, there is insufficient high quality evidence to allow paediatric surgeons, who are responsible for the care of children requiring surgical intervention from birth through to sixteen years of age, to deliver such gold-standard practice. Developing and utilising core outcome sets in research has been proposed as one method through which the quality of the paediatric surgical evidence-base can be improved.
Thesis aim
The overall aim of this thesis was to investigate whether outcomes in paediatric surgery can be improved through the development and use of core outcome sets.
Methods
Core outcome sets were developed for two exemplar paediatric surgical conditions, Hirschsprung’s disease, where the infant’s intestines do not function properly, and gastroschisis, where the infant is born with the intestines outside of the body. The same methodology, consisting of a systematic review, a three-phase Delphi process, a consensus meeting, and a measurement meeting was used for development of each core outcome set. Key stakeholders, including patients, parents of affected children, and clinicians, were involved in each development process.
Subsequently, a joint British Isles-Canadian population-based cohort study was undertaken to investigate the effect of three key treatment modalities; silo repair; operative primary fascial closure; and ward-based reduction; on short-term core outcomes for infants born with gastroschisis. A British-Isles-wide cohort study was also undertaken to describe core outcomes at primary school age for children born with Hirschsprung’s disease. The latter study involved collection of parent reported outcomes as well as data from clinicians.
Findings
The developed Hirschsprung’s disease core outcome set consisted of ten outcomes identified as important by key stakeholders; death, faecal incontinence, bowel function score, unplanned reoperation, voluntary bowel movements, urinary incontinence, psychological stress, quality of life, permanent stoma, and Hirschsprung’s Associated Enterocolitis. Eight outcomes were included in the gastroschisis core outcome set; death, sepsis, growth, number of operations, severe gastrointestinal complication, time on parenteral nutrition, liver disease and quality of life.
In infants born with gastroschisis who had no features of significant bowel injury at birth, the cohort study demonstrated that in comparison to operative primary fascial closure, the use of silo repair was associated with approximately a 75% reduction in the incidence of severe gastrointestinal complications in the first 28 days of life, but at the expense of a 40% increase in number of operations, a doubling in the risk of experiencing one or more infection, and potentially a small increase in number of days on which parenteral nutrition was received over the same period of time.
Lower than anticipated data return rates limited the certainty with which conclusions could be drawn from the Hirschsprung’s disease study. Nevertheless, there was sufficient evidence to suggest that approximately 60% of children with Hirschsprung’s disease are not continent of faeces at five to seven years of age, 45% have undergone at least one unplanned reoperation, and 11% are incontinent of urine. There is also some limited evidence to suggest that one operation used for treating children with Hirschsprung’s disease, the Swenson procedure, is associated with a greater incidence of unplanned reoperations than the other main surgical treatments of Hirschsprung’s disease, the Soave repair and the Duhamel repair.
Conclusions
It is possible, in a practical and expeditious way, to develop core outcome sets for paediatric surgical conditions. It is also possible to use those core outcome sets in large-scale observational studies to address clinically important questions. Results of such studies can be used to improve the care of children undergoing early surgery. The conclusion of this thesis is therefore that developing and implementing the use of core outcome sets can improve outcomes in paediatric surgery.