PMID- 11568898
OWN - NLM
STAT- MEDLINE
DCOM- 20011204
LR  - 20190906
IS  - 0098-1532 (Print)
IS  - 0098-1532 (Linking)
VI  - 37
IP  - 4
DP  - 2001 Oct
TI  - Screening for Wilms tumor and hepatoblastoma in children with Beckwith-Wiedemann 
      syndromes: a cost-effective model.
PG  - 349-56
AB  - BACKGROUND: We undertook a cost-benefit analysis of screening for Wilms tumor and 
      hepatoblastoma in children with Beckwith-Wiedemann syndrome (BWS), a known cancer 
      predisposition syndrome. The purpose of this analysis was twofold: first, to 
      assess whether screening in children with BWS has the potential to be 
      cost-effective; second, if screening appears to be cost-effective, to determine 
      which parameters would be most important to assess if a screening trial were 
      initiated. PROCEDURES: We used data from the BWS registry at the National Cancer 
      Institute, the National Wilms Tumor Study (NWTS), and large published series to 
      model events for two hypothetical cohorts of 1,000 infants born with BWS. One 
      hypothetical cohort was screened for cancer until a predetermined age, 
      representing the base case. The other cohort was unscreened. For our base case, 
      we assumed: (a) sonography examinations three times yearly (triannually) from 
      birth until 7 years of age; (b) screening would result in one stage shift 
      downward at diagnosis for Wilms tumor and hepatoblastoma; (c) 100% sensitivity 
      and 95% specificity for detecting clinical stage I Wilms tumor and 
      hepatoblastoma; (d) a 3% discount rate; (e) a false positive result cost of $402. 
      We estimated mortality rates based on published Wilms tumor and hepatoblastoma 
      stage specific survival. RESULTS: Using the base case, screening a child with BWS 
      from birth until 4 years of age results in a cost per life year saved of $9,642 
      while continuing until 7 years of age results in a cost per life-year saved of 
      $14,740. When variables such as cost of screening examination, discount rate, and 
      effectiveness of screening were varied based on high and low estimates, the 
      incremental cost per life-year saved for screening up until age four remained 
      comparable to acceptable population based cancer screening ranges (< $50,000 per 
      life year saved). CONCLUSIONS: Under our model's assumptions, abdominal 
      sonography examinations in children with BWS represent a reasonable strategy for 
      a cancer screening program. A cancer screening trial is warranted to determine 
      if, when, and how often children with BWS should be screened and to determine 
      cost-effectiveness in clinical practice.
CI  - Published 2001 Wiley-Liss, Inc.
FAU - McNeil, D E
AU  - McNeil DE
AD  - Genetic Epidemiology Branch, National Cancer Institute, National Institutes of 
      Health, 6120 Executive Boulevard, Bethesda, MD 20892-7236, USA.
FAU - Brown, M
AU  - Brown M
FAU - Ching, A
AU  - Ching A
FAU - DeBaun, M R
AU  - DeBaun MR
LA  - eng
GR  - N02 CP 91026/CP/NCI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Med Pediatr Oncol
JT  - Medical and pediatric oncology
JID - 7506654
SB  - IM
MH  - Age Distribution
MH  - Beckwith-Wiedemann Syndrome/diagnostic imaging/*epidemiology
MH  - Child
MH  - Child, Preschool
MH  - Cohort Studies
MH  - Comorbidity
MH  - Cost-Benefit Analysis
MH  - Female
MH  - Hepatoblastoma/diagnostic imaging/*epidemiology
MH  - Humans
MH  - Infant
MH  - Kidney Neoplasms/diagnostic imaging/*epidemiology
MH  - Liver Neoplasms/diagnostic imaging/*epidemiology
MH  - Male
MH  - Mass Screening/*economics
MH  - Registries
MH  - Sensitivity and Specificity
MH  - Sex Distribution
MH  - Survival Analysis
MH  - Ultrasonography
MH  - United States/epidemiology
MH  - Wilms Tumor/diagnostic imaging/*epidemiology
EDAT- 2001/09/25 10:00
MHDA- 2002/01/05 10:01
CRDT- 2001/09/25 10:00
PHST- 2001/09/25 10:00 [pubmed]
PHST- 2002/01/05 10:01 [medline]
PHST- 2001/09/25 10:00 [entrez]
AID - 10.1002/mpo.1209 [pii]
AID - 10.1002/mpo.1209 [doi]
PST - ppublish
SO  - Med Pediatr Oncol. 2001 Oct;37(4):349-56. doi: 10.1002/mpo.1209.