A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease.

Hematopoietic stem cell transplantation Mitochondrial activity Novel protocol on whole blood Phagocytes disorders Primary Immunodeficiency X linked chronic granulomatous disease X linked chronic granulomatous disease carriers strong direct correlation between mitochondrial activity, hematopoietic stem cell transplantation chimerism and DHR monitored before and after transplantation and in XCGD carriers

Journal

Journal of clinical immunology
ISSN: 1573-2592
Titre abrégé: J Clin Immunol
Pays: Netherlands
ID NLM: 8102137

Informations de publication

Date de publication:
11 2022
Historique:
received: 06 02 2022
accepted: 22 07 2022
pubmed: 10 8 2022
medline: 30 11 2022
entrez: 9 8 2022
Statut: ppublish

Résumé

X-linked chronic granulomatous disease is a rare disease caused by mutations in the CYBB gene. While more extensive knowledge is available on genetics, pathogenesis, and possible therapeutic options, mitochondrial activity and its implications on patient monitoring are still not well-characterized. We have developed a novel protocol to study mitochondrial activity on whole blood of XCGD patients before and after transplantation, as well as on XCGD carriers. Here we present results of these analyses and of the restoration of mitochondrial activity in hyperinflamed X-linked Chronic Granulomatous Disease after hematopoietic stem cell transplantation. Moreover, we show a strong direct correlation between mitochondrial activity, chimerism, and DHR monitored before and after transplantation and in XCGD carriers. In conclusion, based on these findings, we suggest testing this new ready-to-use marker to better characterize patients before and after treatment and to investigate disease expression in carriers.

Identifiants

pubmed: 35945378
doi: 10.1007/s10875-022-01338-x
pii: 10.1007/s10875-022-01338-x
pmc: PMC9700633
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1742-1747

Informations de copyright

© 2022. The Author(s).

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Auteurs

Maddalena Migliavacca (M)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Luca Basso Ricci (L)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.

Giada Farinelli (G)

Fondazione Telethon, Milan, Italy.

Valeria Calbi (V)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Francesca Tucci (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Federica Barzaghi (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Francesca Ferrua (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maria Pia Cicalese (MP)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Silvia Darin (S)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.

Lina Raffaella Barzaghi (LR)

Neurosurgery department, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Fabio Giglio (F)

Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Jacopo Peccatori (J)

Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Francesca Fumagalli (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Roberto Nicoletti (R)

Department of Radiology, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Stefania Giannelli (S)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.

Claudia Sartirana (C)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.

Alessandro Bandiera (A)

Thoracic surgery, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maria Esposito (M)

S.O.C. Malattie Infettive ASL VC Piemonte, Milan, Italy.

Raffaella Milani (R)

Cytometry Laboratory, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Benedetta Mazzi (B)

HLA Laboratory, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Andrea Finocchi (A)

Department of Paediatrics, Ospedale Pediatrico Bambino Gesù and University of Rome "Tor Vergata", Rome, Italy.

Sarah Marktel (S)

Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Andrea Assanelli (A)

Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Franco Locatelli (F)

Department of Onco-Haematology and Cell and Gene Therapy, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Department of Pediatrics, Sapienza University of Rome, Rome, Italy.

Fabio Ciceri (F)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Università Vita-Salute San Raffaele, Milan, Italy.

Alessandro Aiuti (A)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy. aiuti.alessandro@hsr.it.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.
Università Vita-Salute San Raffaele, Milan, Italy. aiuti.alessandro@hsr.it.

Maria Ester Bernardo (ME)

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.
Paediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Università Vita-Salute San Raffaele, Milan, Italy.

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