Early Diagnosis and Treatment of Purine Nucleoside Phosphorylase (PNP) Deficiency through TREC-Based Newborn Screening.

T-cell receptor excision circle newborn screening purine nucleoside phosphorylase deficiency severe combined immunodeficiency

Journal

International journal of neonatal screening
ISSN: 2409-515X
Titre abrégé: Int J Neonatal Screen
Pays: Switzerland
ID NLM: 101665400

Informations de publication

Date de publication:
29 Sep 2021
Historique:
received: 22 07 2021
revised: 27 08 2021
accepted: 24 09 2021
entrez: 26 10 2021
pubmed: 27 10 2021
medline: 27 10 2021
Statut: epublish

Résumé

Purine nucleoside phosphorylase (PNP) deficiency is a rare inherited disorder, resulting in severe combined immunodeficiency. To date, PNP deficiency has been detected in newborn screening only through the use of liquid chromatography tandem mass spectrometry. We report the first case in which PNP deficiency was detected by TREC analysis.

Identifiants

pubmed: 34698070
pii: ijns7040062
doi: 10.3390/ijns7040062
pmc: PMC8544499
pii:
doi:

Types de publication

Case Reports

Langues

eng

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Auteurs

Andrea Martín-Nalda (A)

Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Jacques G Rivière (JG)

Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Mireia Català-Besa (M)

Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Marina García-Prat (M)

Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Alba Parra-Martínez (A)

Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Mónica Martínez-Gallo (M)

Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies, 08035 Barcelona, Spain.
Diagnostic Immunology Group, Vall d'Hebron Institut de Recerca (VHIR), Hospital Universitari Vall d'Hebron, Vall d'Hebron Barcelona Hospital Campus, Passeig Vall d'Hebron 119-129, 08035 Barcelona, Spain.
Departament de Biologia Cel·lular, Fisiologia i Immunologia, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.

Roger Colobran (R)

Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies, 08035 Barcelona, Spain.
Diagnostic Immunology Group, Vall d'Hebron Institut de Recerca (VHIR), Hospital Universitari Vall d'Hebron, Vall d'Hebron Barcelona Hospital Campus, Passeig Vall d'Hebron 119-129, 08035 Barcelona, Spain.
Departament de Biologia Cel·lular, Fisiologia i Immunologia, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
Genetics Department, Hospital Universitari Vall d'Hebron, Vall d'Hebron Barcelona Hospital Campus, Passeig Vall d'Hebron 119-129, 08035 Barcelona, Spain.

Ana Argudo-Ramírez (A)

Newborn Screening Laboratory, Inborn Errors of Metabolism Division, Biochemistry and Molecular Genetics Department, Hospital Clínic de Barcelona, 08036 Barcelona, Spain.

Jose Luis Marín-Soria (JL)

Newborn Screening Laboratory, Inborn Errors of Metabolism Division, Biochemistry and Molecular Genetics Department, Hospital Clínic de Barcelona, 08036 Barcelona, Spain.

Judit García-Villoria (J)

Inborn Errors of Metabolism Division, Biochemistry and Molecular Genetics Department, Hospital Clínic de Barcelona, IDIBAPS, CIBERER, 08036 Barcelona, Spain.

Laura Alonso (L)

Pediatric Oncology and Hematology Department, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Jose Antonio Arranz-Amo (JA)

Metabolic Diseases Laboratory, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Giancarlo la Marca (G)

Department of Experimental and Clinical Biomedical Sciences, University of Florence and Newborn Screening, Clinical Chemistry and Pharmacology Lab, Meyer Children's University Hospital, 50139 Florence, Italy.

Pere Soler-Palacín (P)

Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.
Immunology Division, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, 08035 Barcelona, Spain.

Classifications MeSH