title: An economic evaluation of pediatric small bowel transplantation in the United Kingdom.
abstract: BACKGROUND
  Small bowel transplantation (SBTx) offers an alternative to parenteral nutrition (PN) for the treatment of chronic intestinal failure in children: this study estimated its cost-effectiveness in the early phase of a U.K. program.
  
  
  METHODS
  Children assessed for SBTx were categorized as: 1) requiring SBTx following PN-related complications (n=23), 2) stable at home not requiring SBTx (n=24), and 3) terminally ill and unsuitable for SBTx (n=6). Costs were estimated from detailed resource-use data. Two comparisons were used for effectiveness: actual survival following transplantation (n=14) compared to: 1) estimated survival without transplantation using a prognostic model, and 2) the waiting list experiences of all patients listed for SBTx (n=23).
  
  
  RESULTS
  Mean costs up to 30 months were pounds sterling 207,000 for those transplanted or on the waiting list, pounds sterling 159,000 for those stable on home PN, and pounds sterling 56,000 for those terminally ill. The prognostic model estimated a mean survival gain from transplantation of 0.12 years over 30 months, and suggested that transplantation was cost-saving. The second approach suggested that transplantation reduced survival by 0.24 years at an additional cost of pounds sterling 131,000.
  
  
  CONCLUSIONS
  Firm conclusions on cost-effectiveness of SBTx are not possible given the two different estimates. The prognostic model approach (suggesting that pediatric SBTx may provide a small survival benefit at a small reduction in costs) should be less subject to bias, but the model requires external validation. Meanwhile, children at risk of fatal PN-complications should be given the opportunity to receive a SBTx only within a continuing formal assessment of the technology.
authors.affiliation: Health Economics Research Group, Brunel University, Uxbridge, Middlesex, United Kingdom.
authors.forename: Louise
authors.initials: L
authors.lastname: Longworth
authors.forename: Tracey
authors.initials: T
authors.lastname: Young
authors.forename: Sue V
authors.initials: SV
authors.lastname: Beath
authors.forename: Deirdre A
authors.initials: DA
authors.lastname: Kelly
authors.forename: Hema
authors.initials: H
authors.lastname: Mistry
authors.forename: Sue M
authors.initials: SM
authors.lastname: Protheroe
authors.forename: Julie
authors.initials: J
authors.lastname: Ratcliffe
authors.forename: Martin J
authors.initials: MJ
authors.lastname: Buxton
mesh_terms: D002648:Child; D002675:Child, Preschool; D003362:Cost-Benefit Analysis; D005260:Female; D006801:Humans; D007223:Infant; D007421:Intestine, Small / Q000637:transplantation*; D008297:Male; D016331:Parenteral Nutrition, Home; D011379:Prognosis