A population-based nested case control study on recurrent pneumonias in children with severe generalized cerebral palsy: ethical considerations of the design and representativeness of the study sample

Springer Science and Business Media LLC - 2005
Rebekka Veugelers1,2, Elsbeth A.C. Calis2,1, Corine Penning1,2, Arianne Verhagen3, Roos Bernsen3, J. Bouquet4, Marc A. Benninga5, Peter Merkus6, Hubertus G.M. Arets7, Dick Tibboel2, Heleen M. Evenhuis1
1Intellectual Disability Medicine, department of General Practice Erasmus MC, Rotterdam, The Netherlands
2Department of Paediatric Surgery, Erasmus MC, Sophia Children's Hospital, Rotterdam, The Netherlands
3Department of General Practice, Erasmus MC, Rotterdam, The Netherlands
4Department of Paediatric Gastro-enterology Erasmus MC, Rotterdam, The Netherlands
5Department of Paediatric Gastro-enterology and Nutrition Academic Medical Centre / Emma's Children's Hospital, Amsterdam, The Netherlands
6Department of Paediatric Pulmonology, Erasmus MC, Rotterdam, The Netherlands
7Department of Paediatric Pulmonology UMC, HP KH.01.419.0, Utrecht, The Netherlands

Tóm tắt

AbstractBackgroundIn children with severe generalized cerebral palsy, pneumonias are a major health issue. Malnutrition, dysphagia, gastro-oesophageal reflux, impaired respiratory function and constipation are hypothesized risk factors. Still, no data are available on the relative contribution of these possible risk factors in the described population. This paper describes the initiation of a study in 194 children with severe generalized cerebral palsy, on the prevalence and on the impact of these hypothesized risk factors of recurrent pneumonias.Methods/DesignA nested case-control design with 18 months follow-up was chosen. Dysphagia, respiratory function and constipation will be assessed at baseline, malnutrition and gastro-oesophageal reflux at the end of the follow-up. The study population consists of a representative population sample of children with severe generalized cerebral palsy. Inclusion was done through care-centres in a predefined geographical area and not through hospitals. All measurements will be done on-site which sets high demands on all measurements. If these demands were not met in "gold standard" methods, other methods were chosen. Although the inclusion period was prolonged, the desired sample size of 300 children was not met. With a consent rate of 33%, nearly 10% of all eligible children in the Netherlands are included (n = 194). The study population is subtly different from the non-participants with regard to severity of dysphagia and prevalence rates of pneumonias and gastro-oesophageal reflux.DiscussionEthical issues complicated the study design. Assessment of malnutrition and gastro-oesophageal reflux at baseline was considered unethical, since these conditions can be easily treated. Therefore, we postponed these diagnostics until the end of the follow-up. In order to include a representative sample, all eligible children in a predefined geographical area had to be contacted. To increase the consent rate, on-site measurements are of first choice, but timely inclusion is jeopardised. The initiation of this first study among children with severe neurological impairment led to specific, unexpected problems. Despite small differences between participants and non-participating children, our sample is as representative as can be expected from any population-based study and will provide important, new information to bring us further towards effective interventions to prevent pneumonias in this population.

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