Background: Children with multicystic kidney disease (MCKD) are increasingly managed conservatively and are followed up throughout childhood because they are perceived to be at increased risk of developing Wilms' tumour. With this risk still poorly defined and somewhat controversial, the strategy and the duration of follow up do not seem to be based on evidence.
Methods: Systematic review of the literature for all published cohort studies (prospective and retrospective) of children diagnosed to have unilateral MCKD and managed conservatively.
Exclusion criteria: bilateral MCKD, nephrectomy (not for malignancy) during the follow up period. We estimated for children with MCKD the probability of developing Wilms' tumour during the follow up period, with 95% CI using the Poisson distribution.
Results: From 26 reviewed studies, no cases of Wilms' tumour developed in 1041 eligible children. The mean probability of a child with unilateral MCKD to develop Wilms' was therefore nil, with a 97.5% upper CI estimated at 0.0035 (or 3.5 per 1000 children).
Conclusion: The development of a national or a European registry for children with MCKD would increase the precision of their risk estimate to develop Wilms' tumour. In the meantime, there is no evidence to support any of the different modalities for following up these children by ultrasound, if indeed such a strategy is necessary.