Evidence map›Paper›PMID 28270365›Full record

ArticleThe Journal of allergy and clinical immunology2017

Treatment of infants identified as having severe combined immunodeficiency by means of newborn screening.

Morna J Dorsey, Christopher C Dvorak, Morton J Cowan, Jennifer M Puck

Registry-linked trialOpen access · greenAbstract read
In one paragraph

Article in The Journal of allergy and clinical immunology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03538899 (Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency), which is not on this map. Cited by 41 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed, 2 pooled it
4.6field-weighted citation impact, top 4% 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.

NCT03538899 phase1 / phase2recruitingnot on this mapstarted 2018, after this paper: background citation

Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells

TypeinterventionalSponsorUniversity of California, San FranciscoRan2018 to 2038Enrolled24ConditionsSevere Combined ImmunodeficiencyArmsAProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
3 · Its place in the literature

Who cites it

41 citing papers in PubMed, 2 syntheses or guidelines pooled it, 80 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. Genetically-determined defects of T cell development.Allergy and asthma proceedings · 2024
    Review
  9. Article
  10. Article
  11. Review
  12. Review
  13. Review
  14. Article
  15. Future Perspectives of Newborn Screening for Inborn Errors of Immunity.International journal of neonatal screening · 2021
    Review
  16. Article
  17. Article
  18. Article
  19. Review
  20. Impact of maternal engrafted cytomegalovirus-specific CD8Clinical & translational immunology · 2021
    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

4 authors at 1 institution in 1 country.

Morna J DorseyDepartment of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplantation, University of California, San Francisco, Calif. Electronic address: morna.dorsey@ucsf.edu.
Christopher C DvorakDepartment of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplantation, University of California, San Francisco, Calif.
Morton J CowanDepartment of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplantation, University of California, San Francisco, Calif.
Jennifer M PuckDepartment of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplantation, University of California, San Francisco, Calif.
University of California, San Francisco · US

Funding

Engaging University of California Stakeholders for Biorespository ResearchUL1TR000004 · NCATS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI GRANDIS, JENNIFER RUBIN · 2012 to 2015
$78.0M
Training of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI TORGERSON, TROY R · 2009 to 2023
$23.3M
Newborn Screening for T-Cell Disorders: Spectrum and OutcomesR01AI105776 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BRENNER, STEVEN E, CHURCH, JOSEPH · 2013 to 2016
$2.4M
Primary Immune Deficiency Treatment Consortium Annual Scientific MeetingR13AI094943 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2011 to 2025
$239k
NCATS NIH HHS UL1 TR000004NIAID NIH HHS R01 AI105776NIAID NIH HHS R13 AI094943NIAID NIH HHS U54 AI082973
6 · The paper itself

Abstract

Severe combined immunodeficiency (SCID) is characterized by severely impaired T-cell development and is fatal without treatment. Newborn screening (NBS) for SCID permits identification of affected infants before development of opportunistic infections and other complications. Substantial variation exists between treatment centers with regard to pretransplantation care, and transplantation protocols for NBS identified infants with SCID, as well as infants with other T-lymphopenic disorders detected by using NBS. We developed approaches to management based on the study of infants identified by means of NBS for SCID who received care at the University of California, San Francisco (UCSF). From August 2010 through October 2016, 32 patients with NBS-identified SCID and leaky SCID from California and other states were treated, and 42 patients with NBS-identified non-SCID T-cell lymphopenia were followed. Our center's approach supports successful outcomes; systematic review of our practice provides a framework for diagnosis and management, recognizing that more data will continue to shape best practices.

Indexed as

Neonatal ScreeningHumansInfant, NewbornParentsSevere Combined Immunodeficiencynewborn screeningSevere combined immunodeficiencyT-cell lymphopeniaT cellstransplantation

Identifiers

PMID28270365
PMCPMC5385855
OpenAlexW2592164063

What OpenQuestion holds

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Read underepoch 390

Registered trials

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.