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Screening for Wilms' tumor in children with high-risk congenital syndromes: considerations for an intervention trial
M R DeBaun1, M Brown, L Kessler
1Genetic Epidemiology Branch, National Cancer Institute, National Institutes of Health, Rockville, Maryland 20892-7372, USA.
Insights
Screening children with specific congenital syndromes for Wilms' tumor using renal sonography may be effective. Cost-effectiveness analysis is crucial before initiating a randomized trial for this high-risk pediatric population.
Area of Science:
- Pediatric oncology
- Medical screening technologies
- Congenital syndromes
Background:
- Cancer screening in children is rare, but certain congenital syndromes increase Wilms' tumor risk.
- These children are identifiable, have high tumor incidence, and may benefit from early detection.
- Renal sonography is a feasible screening tool for this population.
Purpose of the Study:
- To review conditions for a successful screening trial in children at high risk for Wilms' tumor.
- To discuss the role of cost-effectiveness analysis in evaluating screening interventions.
- To identify key parameters for assessing the cost and effectiveness of Wilms' tumor screening.
Main Methods:
- Review of existing literature on Wilms' tumor screening in at-risk pediatric populations.
- Discussion of criteria for designing a randomized screening trial.
- Exploration of cost-effectiveness analysis as a preliminary evaluation tool.
Main Results:
- Children with specific congenital syndromes represent a viable group for Wilms' tumor screening.
- Cost and effectiveness parameters need systematic evaluation before a randomized trial.
- Simulation models like cost-effectiveness analysis can guide screening decisions.
Conclusions:
- Screening for Wilms' tumor in children with associated congenital syndromes warrants further investigation.
- Cost-effectiveness analysis is a valuable preliminary step to determine the feasibility of screening trials.
- Identifying optimal screening conditions is essential for potential future randomized trials.
Abstract:
Screening for cancer in children is uncommon. However, in children with congenital syndromes associated with Wilms' tumor, conditions exist that potentially make screening effective. This select population of children 1) are relatively easily identified; 2) have a high incidence of Wilms' tumor; 3) if identified before development of Wilms' tumor, may have a decrement in morbidity/mortality; and 4) are amenable to a simple and acceptable screening technology, renal sonography exams. Many clinicians have recommended screening for cancer in children with congenital syndromes associated with Wilms' tumor. However, neither costs nor effectiveness of such recommendations have been evaluated systematically. The strongest evidence for or against Wilms' tumor screening in this select population would be provided by a randomized screening trial. Prior to undertaking such a trial, the key parameters that dominate the cost and effectiveness of screening should be identified. Simulation models, such as cost effectiveness analysis, offer a starting point for deciding whether cancer screening is appropriate, and if so, under what set of conditions. We review basic conditions required for a successful screening trial in children with syndromes that are at increased risk of Wilms' tumor. We also discuss the use of cost-effectiveness analysis as a preliminary step in determining the feasibility of an intervention trial.