Evidence map›Paper›PMID 30805731›Full record

ArticleEuropean journal of pediatrics2019

Cost-effectiveness of newborn screening for severe combined immunodeficiency.

Catharina P B Van der Ploeg, Maartje Blom, Robbert G M Bredius, Mirjam van der Burg, Peter C J I Schielen, Paul H Verkerk, M Elske Van den Akker-van Marle

Abstract read
PubMed Publisher
In one paragraph

Article in European journal of pediatrics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed, 2 pooled it
1.4field-weighted citation impact, top 19% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

22 citing papers in PubMed, 2 syntheses or guidelines pooled it, 27 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Article
  4. Review
  5. Article
  6. Article
  7. Review
  8. Article
  9. Review
  10. Article
  11. Review
  12. Article
  13. Article
  14. Newborn Screening by Genomic Sequencing: Opportunities and Challenges.International journal of neonatal screening · 2022
    Article
  15. Article
  16. Article
  17. Article
  18. Achieving the Quadruple Aim to deliver value-based allergy care in an ever-evolving health care system.Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology · 2020
    Review
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 3 institutions in 1 country.

Catharina P B Van der PloegTNO - Child Health, PO box 3005, 2301 DA, Leiden, The Netherlands. Kitty.vanderPloeg@tno.nl.ORCID http://orcid.org/0000-0002-5393-8938
Maartje BlomDepartment of Pediatrics, Leiden University Medical Center, PO box 9600, 2300 RC, Leiden, The Netherlands.
Robbert G M BrediusDepartment of Pediatrics, Leiden University Medical Center, PO box 9600, 2300 RC, Leiden, The Netherlands.
Mirjam van der BurgDepartment of Pediatrics, Leiden University Medical Center, PO box 9600, 2300 RC, Leiden, The Netherlands.
Peter C J I SchielenRIVM, Department Biologicals, Screening and Innovation, PO box 1, 3720 BA, Bilthoven, The Netherlands.
Paul H VerkerkTNO - Child Health, PO box 3005, 2301 DA, Leiden, The Netherlands.
M Elske Van den Akker-van MarleDepartment of Medical Decision Making, Leiden University Medical Center, PO box 9600, 2300 RC, Leiden, The Netherlands.
Leiden University Medical Center · NLNational Institute for Public Health and the Environment · NLNetherlands Organisation for Applied Scientific Research · NL

Funding

Rijksinstituut voor Volksgezondheid en Milieu 3910086068
6 · The paper itself

Abstract

Severe combined immunodeficiency (SCID) is a condition that often results in severe infections and death at young age. Early detection shortly after birth, followed by treatment before infections occur, largely increases the chances of survival. As the incidence of SCID is low, assessing cost-effectiveness of adding screening for SCID to the newborn screening program is relevant for decision making. Lifetime costs and effects of newborn screening for SCID were compared to a situation without screening in the Netherlands in a decision analysis model. Model parameters were based on literature and expert opinions. Sensitivity analyses were performed. Due to earlier detection, the number of deaths due to SCID per 100,000 children was assessed to decrease from 0.57 to 0.23 and a number of 11.7 quality adjusted life-years (QALYs) gained was expected. Total yearly healthcare costs, including costs of screening, diagnostics, and treatment, were €390,800 higher in a situation with screening compared to a situation without screening, resulting in a cost-utility ratio of €33,400 per QALY gained.Conclusion: Newborn screening for SCID might be cost-effective. However, there is still a lot of uncertainty around the cost-effectiveness estimate. Pilot screening projects are warranted to obtain more accurate estimates for the European situation. What is Known: • Severe combined immunodeficiency (SCID) is a condition that often results in severe infections and death at a young age. • As the incidence of SCID is low, assessing cost-effectiveness of adding screening for SCID to the newborn screening program is needed. What is New: • Newborn screening for SCID is expected to reduce mortality from 0.57 to 0.23 per 100,000 children at additional healthcare costs of €390,800. The cost-utility ratio is €33,400 per QALY gained. • Due to large uncertainty around cost-effectiveness estimates, pilot screening projects are warranted for Europe.

Indexed as

Cost-Benefit AnalysisHealth Care CostsHumansInfant, NewbornNeonatal ScreeningNetherlandsQuality-Adjusted Life YearsSevere Combined ImmunodeficiencyCost-effectiveness analysisDecision analysis modelNewborn screeningSevere combined immunodeficiency

Identifiers

PMID30805731
OpenAlexW2915353766

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.