Publication Cover
Acta Clinica Belgica
International Journal of Clinical and Laboratory Medicine
Volume 75, 2020 - Issue 3
323
Views
7
CrossRef citations to date
0
Altmetric
Articles

A model-based economic evaluation of four newborn screening strategies for cystic fibrosis in Flanders, Belgium

ORCID Icon, ORCID Icon, ORCID Icon, , , & ORCID Icon show all

References

  • Brown SD, White R, Tobin P. Keep them breathing: cystic fibrosis pathophysiology, diagnosis, and treatment. JAAPA. 2017;30(5):23–27.
  • Castellani C, Southern KW, Brownlee K, et al. European best practice guidelines for cystic fibrosis neonatal screening. J Cyst Fibros. 2009;8(3):153–173.
  • Barben J, Castellani C, Dankert-Roelse J, et al. The expansion and performance of national newborn screening programmes for cystic fibrosis in Europe. J Cyst Fibros. 2017;16(2):207–213.
  • Munck A, Mayell SJ, Winters V, et al. Cystic Fibrosis Screen Positive, Inconclusive Diagnosis (CFSPID): A new designation and management recommendations for infants with an inconclusive diagnosis following newborn screening. J Cyst Fibros. 2015;14(6):706–713.
  • Barben J, Southern KW. Cystic fibrosis screen positive, inconclusive diagnosis. Curr Opin Pulm Med. 2016;22(6):617–622.
  • Ren CL, Borowitz DS, Gonska T, et al. Cystic fibrosis transmembrane conductance regulator-related metabolic syndrome and cystic fibrosis screen positive, inconclusive diagnosis. J Pediatr. 2017;181s:S45–S51.e1.
  • Schmidt M, Werbrouck A, Verhaeghe N, et al. Strategies for newborn screening for cystic fibrosis: A systematic review of health economic evaluations. J Cyst Fibros. 2018;17:306–315.
  • Drummond M, Barbieri M, Cook J, et al. Transferability of economic evaluations across jurisdictions: ISPOR Good Research Practices Task Force report. Value Health. 2009;12(4):409–418.
  • Wells J, Rosenberg M, Hoffman G, et al. A decision-tree approach to cost comparison of newborn screening strategies for cystic fibrosis. Pediatrics. 2012;129(2):e339–e347.
  • Van der Ploeg C, Van den Akker-van Marle M, Vernooij-van Langen A, et al. Costeffectiveness of newborn screening for cystic fibrosis determined with real-life data. J Cyst Fibros. 2015;14(2):194–202.
  • Rasch A, Perleth M. Modellierung der kurzfristigen diagnostischen und ökonomischen Auswirkungen eines Neugeborenen-screenings auf Mukoviszidose [A short-term diagnostic and economic impact model of neonatal screening for cystic fibrosis]. Klin Padiatr. 2011;223(2):96–103.
  • Seror V, Cao C, Roussey M, et al. PAP assays in newborn screening for cystic fibrosis: a population-based cost-effectiveness study. J Med Screen. 2016;23(2):62–69.
  • Haute Autorité de Santé (HAS). Place de la stratégie couplant les dosages de la trypsine immunoréactive (TIR) et de la protéine associée à la pancréatite (PAP) dans le dépistage systématique de la mucoviscidose en France [Role of the strategy coupling immunoreactive trypsin (TIR) and pancreatitis-associated protein (PAP) assays in routine screening for cystic fibrosis in France]. Saint-Denis: HAS; 2015.
  • Nshimyumukiza L, Bois A, Daigneault P, et al. Cost effectiveness of newborn screening for cystic fibrosis: a simulation study. J Cyst Fibros. 2014;13(3):267–274.
  • Munck A, Roussey M. Le dépistage néonatal de la mucoviscidose: stratégie et résultats nationaux [The French nationwide cystic fibrosis newborn screening program: strategy and results]. Arch Pediatr. 2008;15:S1–S6.
  • Sommerburg O, Krulisova V, Hammermann J, et al. Comparison of different IRTPAP protocols to screen newborns for cystic fibrosis in three central European populations. J Cyst Fibros. 2014;13(1):15–23.
  • Sommerburg O, Stahl M, Hammermann J, et al. Neugeborenenscreening auf Mukoviszidose in Deutschland: vergleich des neuen Screening-Protokolls mit einem Alternativprotokoll [Newborn Screening on Cystic Fibrosis in Germany: comparison of the new Screening Protocol with an Alternative Protocol]. KlinPadiatr. 2017;229(2):59–66.
  • Rijksinstituut voor Volksgezondheid en Milieu (RIVM). Afkapgrenzen en beslissingscriteria neonatale screening [Cut-off limits and decision criteria neonatal screening]. Bilthoven: RIVM; 2017. Version 1 January 2017.
  • Kloosterboer M, Hoffman G, Rock M, et al. Clarification of laboratory and clinical variables that influence cystic fibrosis newborn screening with initial analysis of immunoreactive trypsinogen. Pediatrics. 2009;123(2):e338–e346.
  • Therrell BL, Hannon WH, Hoffman G, et al. Immunoreactive trypsinogen (IRT) as a biomarker for cystic fibrosis: challenges in newborn dried blood spot screening. Mol Genet Metab. 2012;106(1):1–6.
  • Castellani C, Massie J, Sontag M, et al. Newborn screening for cystic fibrosis. Lancet Respir Med. 2016;4(8):653–661.
  • Vernooij-van Langen AM, Loeber JG, Elvers B, et al. Novel strategies in newborn screening for cystic fibrosis: a prospective controlled study. Thorax. 2012;67(4):289–295.
  • Van den Akker-van Marle ME, Dankert HM, Verkerk PH, et al. Cost-effectiveness of 4 neonatal screening strategies for cystic fibrosis. Pediatrics. 2006;118(3):896–905.
  • Hammond KB, Abman SH, Sokol RJ, et al. Efficacy of statewide neonatal screening for cystic fibrosis by assay of trypsinogen concentrations. N Engl J Med. 1991;325(11):769–774.
  • Ryley H, Deam S, Williams J, et al. Neonatal screening for cystic fibrosis in Wales and the West Midlands: 1. Evaluation of immunoreactive trypsin test. J Clin Pathol. 1988;41(7):726–729.
  • Sommerburg O, Lindner M, Muckenthaler M, et al. Initial evaluation of a biochemical cystic fibrosis newborn screening by sequential analysis of immunoreactive trypsinogen and pancreatitis-associated protein (IRT/PAP) as a strategy that does not involve DNA testing in a Northern European population. J Inherit Metab Dis. 2010;33(2):263–271.
  • Sarles J, Berthezene P, Le Louarn C, et al. Combining immunoreactive trypsinogen and pancreatitis-associated protein assays, a method of newborn screening for cystic fibrosis that avoids DNA analysis. J Pediatr. 2005;147(3):302–305.
  • Belgian Health Care Knowledge Center (KCE). Is neonatale screening op mucoviscidose aangewezen in België? Health technologie assessment (HTA) [Cystic fibrosis neonatal screening health technology assessment (HTA)]. Brussels: KCE; 2010. KCE reports 132A. D/2010/10.273/41.
  • CCEMG–EPPI-Centre Cost Converter [Internet]. London (UK); 2016 [cited 2018 Jan 23]. Available from: https://eppi.ioe.ac.uk/costconversion/.
  • Drummond MF, Sculpher MJ, Claxton K, et al. Methods for the economic evaluation of health care programmes. 4th. New York (NY): Oxford University Press; 2015.
  • Briggs A, Sculpher M, Claxton K. Decision modelling for health economic evaluation. New York (NY): Oxford University Press; 2006.
  • Mayell SJ, Munck A, Craig JV, et al. A European consensus for the evaluation and management of infants with an equivocal diagnosis following newborn screening for cystic fibrosis. J Cyst Fibros. 2009;8(1):71–78.
  • Ozdemir TR, Kanik A. A case of cystic fibrosis with a rare mutation (3849+ 10 kbC> T) and normal sweat chloride levels. Iran J Pediatr. 2015;25(2):e369.
  • Belgian Health Care Knowledge Center (KCE), Belgian guidelines for economic evaluations and budget impact analyses. 2nd. ( KCE Report 183C. D/2012/10.273/54). KCE: Brussels, 2015.
  • Geboorten en Vruchtbaarheid 2015 (Voorlopige cijfers). [Births and Fertility 2015 (Provisional figures)]. Brussels: Statbel; 2015 cited 2018 Jan 23. Available from: https://statbel.fgov.be/nl/themas/bevolking/geboorten-en-vruchtbaarheid#figures
  • Belgian Cystic Fibrosis Registry (BCFR). Jaarlijks Rapport Belgisch Mucoviscidose Register 2013 [Annual Report Belgian Cystic Fibrosis Registry 2013]. Brussels: BCFR; 2015. D/2015/2505/32.
  • Mucoviscidose [Cystic Fibrosis]. Brussels: UZ Brussel; 2018. cited 2018 Jan 23; Available from: http://www.uzbrussel.be/u/view/nl/6710516-Mucoviscidose.html

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.