Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.

enzyme replacement therapy gene therapy isoniazid newborn screening rifampin tuberculosis

Journal

Biomedicines
ISSN: 2227-9059
Titre abrégé: Biomedicines
Pays: Switzerland
ID NLM: 101691304

Informations de publication

Date de publication:
24 Jun 2023
Historique:
received: 15 05 2023
revised: 20 06 2023
accepted: 21 06 2023
medline: 29 7 2023
pubmed: 29 7 2023
entrez: 29 7 2023
Statut: epublish

Résumé

Vaccination with Bacillus Calmette-Guérin (BCG) can be harmful to patients with combined primary immunodeficiencies. We report the outcome of BCG vaccination in a series of twelve patients affected by adenosine deaminase deficiency (ADA-SCID). BCG vaccination resulted in a very high incidence of complications due to uncontrolled replication of the mycobacterium. All patients who developed BCG-related disease were treated successfully and remained free from recurrence of disease. We recommend the prompt initiation of enzyme replacement therapy and secondary prophylaxis to reduce the risk of BCG-related complications in ADA-SCID patients.

Identifiants

pubmed: 37509449
pii: biomedicines11071809
doi: 10.3390/biomedicines11071809
pmc: PMC10376767
pii:
doi:

Types de publication

Journal Article

Langues

eng

Subventions

Organisme : Telethon Foundation
ID : institutional core grant

Références

Blood. 2022 Aug 18;140(7):685-705
pubmed: 35671392
J Allergy Clin Immunol. 2014 Apr;133(4):1134-41
pubmed: 24679470
N Engl J Med. 2009 Jan 29;360(5):447-58
pubmed: 19179314
Haematologica. 2021 Feb 01;106(2):641-646
pubmed: 32817285
Nat Commun. 2022 Mar 14;13(1):1315
pubmed: 35288539
Wkly Epidemiol Rec. 2018 Feb 23;93(8):73-96
pubmed: 29474026
Blood. 2016 Jul 7;128(1):45-54
pubmed: 27129325
BMJ Open. 2022 Nov 21;12(11):e063872
pubmed: 36410811
J Allergy Clin Immunol. 2021 Feb;147(2):417-426
pubmed: 33551023
J Clin Immunol. 2020 May;40(4):625-636
pubmed: 32377975
Lancet Infect Dis. 2017 Mar;17(3):285-295
pubmed: 27964822
Clin Infect Dis. 2006 Feb 15;42(4):548-58
pubmed: 16421800
BMJ Glob Health. 2022 Jan;7(1):
pubmed: 35039309
Lancet Glob Health. 2022 Feb;10(2):e207-e215
pubmed: 34895517
PLoS Med. 2011 Mar;8(3):e1001012
pubmed: 21445325
Lancet Glob Health. 2022 Sep;10(9):e1307-e1316
pubmed: 35961354
J Clin Immunol. 2022 Jan;42(1):195-198
pubmed: 34622389
Haematologica. 2015 Jul;100(7):978-88
pubmed: 26022711
N Engl J Med. 2019 Mar 14;380(11):1001-1011
pubmed: 30865794

Auteurs

Daniele Canarutto (D)

Faculty of Medicine and Surgery, Vita-Salute S. Raffaele University, 20132 Milan, Italy.
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Chiara Oltolini (C)

Clinic of Infectious Diseases, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Federica Barzaghi (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Valeria Calbi (V)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Maddalena Migliavacca (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Francesca Tucci (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Vera Gallo (V)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Giulia Consiglieri (G)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Francesca Ferrua (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Salvatore Recupero (S)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Maria Celia Cervi (MC)

Pediatric Infectious Diseases Division, Department of Pediatrics, Ribeirao Preto Medical School, University of Sao Paulo, Sao Paulo 05508-000, Brazil.

Hamoud Al-Mousa (H)

Department of Pediatrics, King Faisal Specialist Hospital and Research Center, Riyadh 11564, Saudi Arabia.

Anna Pituch-Noworolska (A)

Department of Immunology, University Children's Hospital, 30-663 Krakow, Poland.

Chiara Tassan Din (C)

Clinic of Infectious Diseases, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Paolo Scarpellini (P)

Clinic of Infectious Diseases, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Paolo Silvani (P)

Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Claudia Fossati (C)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Miriam Casiraghi (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Daniela Maria Cirillo (DM)

Emerging Bacterial Pathogens Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Antonella Castagna (A)

Faculty of Medicine and Surgery, Vita-Salute S. Raffaele University, 20132 Milan, Italy.
Clinic of Infectious Diseases, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Maria Ester Bernardo (ME)

Faculty of Medicine and Surgery, Vita-Salute S. Raffaele University, 20132 Milan, Italy.
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Alessandro Aiuti (A)

Faculty of Medicine and Surgery, Vita-Salute S. Raffaele University, 20132 Milan, Italy.
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Maria Pia Cicalese (MP)

Faculty of Medicine and Surgery, Vita-Salute S. Raffaele University, 20132 Milan, Italy.
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.

Classifications MeSH