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Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction

Mesenchymal stromal cells (MSCs) have been proposed as a new therapeutic strategy to treat congenital and acquired respiratory system diseases. We describe a case report of an 18-month-old male patient with progressive chronic respiratory failure, associated with mutations of the surfactant protein...

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Autores principales: Pelizzo, Gloria, Avanzini, Maria Antonietta, Croce, Stefania, Mandelli, Anna, Lenta, Elisa, Farolfi, Andrea, Valsecchi, Chiara, Zirpoli, Salvatore, Lanfranchi, Giulia, Durante, Eleonora, Zoia, Elena, Zuccotti, Gianvincenzo, Calcaterra, Valeria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9857592/
https://www.ncbi.nlm.nih.gov/pubmed/36670712
http://dx.doi.org/10.3390/children10010162
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author Pelizzo, Gloria
Avanzini, Maria Antonietta
Croce, Stefania
Mandelli, Anna
Lenta, Elisa
Farolfi, Andrea
Valsecchi, Chiara
Zirpoli, Salvatore
Lanfranchi, Giulia
Durante, Eleonora
Zoia, Elena
Zuccotti, Gianvincenzo
Calcaterra, Valeria
author_facet Pelizzo, Gloria
Avanzini, Maria Antonietta
Croce, Stefania
Mandelli, Anna
Lenta, Elisa
Farolfi, Andrea
Valsecchi, Chiara
Zirpoli, Salvatore
Lanfranchi, Giulia
Durante, Eleonora
Zoia, Elena
Zuccotti, Gianvincenzo
Calcaterra, Valeria
author_sort Pelizzo, Gloria
collection PubMed
description Mesenchymal stromal cells (MSCs) have been proposed as a new therapeutic strategy to treat congenital and acquired respiratory system diseases. We describe a case report of an 18-month-old male patient with progressive chronic respiratory failure, associated with mutations of the surfactant protein C gene (SFTPC) due to c.289G > T variant p.Gly97Ser (rs927644577) and c.176A > G variant (p.His59Arg), submitted to repeated intravenous infusions of allogeneic bone marrow (BM) MSCs. The clinical condition of the patient was monitored. Immunologic studies before and during MSC treatment were performed. No adverse events related to the MSC infusions were recorded. Throughout the MSC treatment period, the patient showed a growth recovery. Starting the second infusion, the patient experienced an improvement in his respiratory condition, with progressive adaptation to mechanical ventilation. After the third infusion, five hours/die of spontaneous breathing was shown, and after infusion IV, spontaneous ventilation for 24/24 h was recorded. A gradual decrease of lymphocytes and cell subpopulations was observed. No variations in the in vitro T cell response to PHA were determined by MSC treatment as well as for the in vitro B cell response. A decrease in IFN-γ, TNF-α, and IL-10 levels was also detected. Even though we cannot exclude an improvement of pulmonary function due to the physiological maturation, the well-known action of MSCs in the repair of lung tissue, together with the sequence of events observed in our patient, may support the therapeutic role of MSCs in this clinical condition. However, further investigations are necessary to confirm the result and long-term follow-up will be mandatory to confirm the benefits on the pulmonary condition.
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spelling pubmed-98575922023-01-21 Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction Pelizzo, Gloria Avanzini, Maria Antonietta Croce, Stefania Mandelli, Anna Lenta, Elisa Farolfi, Andrea Valsecchi, Chiara Zirpoli, Salvatore Lanfranchi, Giulia Durante, Eleonora Zoia, Elena Zuccotti, Gianvincenzo Calcaterra, Valeria Children (Basel) Article Mesenchymal stromal cells (MSCs) have been proposed as a new therapeutic strategy to treat congenital and acquired respiratory system diseases. We describe a case report of an 18-month-old male patient with progressive chronic respiratory failure, associated with mutations of the surfactant protein C gene (SFTPC) due to c.289G > T variant p.Gly97Ser (rs927644577) and c.176A > G variant (p.His59Arg), submitted to repeated intravenous infusions of allogeneic bone marrow (BM) MSCs. The clinical condition of the patient was monitored. Immunologic studies before and during MSC treatment were performed. No adverse events related to the MSC infusions were recorded. Throughout the MSC treatment period, the patient showed a growth recovery. Starting the second infusion, the patient experienced an improvement in his respiratory condition, with progressive adaptation to mechanical ventilation. After the third infusion, five hours/die of spontaneous breathing was shown, and after infusion IV, spontaneous ventilation for 24/24 h was recorded. A gradual decrease of lymphocytes and cell subpopulations was observed. No variations in the in vitro T cell response to PHA were determined by MSC treatment as well as for the in vitro B cell response. A decrease in IFN-γ, TNF-α, and IL-10 levels was also detected. Even though we cannot exclude an improvement of pulmonary function due to the physiological maturation, the well-known action of MSCs in the repair of lung tissue, together with the sequence of events observed in our patient, may support the therapeutic role of MSCs in this clinical condition. However, further investigations are necessary to confirm the result and long-term follow-up will be mandatory to confirm the benefits on the pulmonary condition. MDPI 2023-01-14 /pmc/articles/PMC9857592/ /pubmed/36670712 http://dx.doi.org/10.3390/children10010162 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Pelizzo, Gloria
Avanzini, Maria Antonietta
Croce, Stefania
Mandelli, Anna
Lenta, Elisa
Farolfi, Andrea
Valsecchi, Chiara
Zirpoli, Salvatore
Lanfranchi, Giulia
Durante, Eleonora
Zoia, Elena
Zuccotti, Gianvincenzo
Calcaterra, Valeria
Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction
title Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction
title_full Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction
title_fullStr Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction
title_full_unstemmed Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction
title_short Allogeneic Mesenchymal Stromal Cells as a Global Pediatric Prospective Approach in the Treatment of Respiratory Failure Associated with Surfactant Protein C Dysfunction
title_sort allogeneic mesenchymal stromal cells as a global pediatric prospective approach in the treatment of respiratory failure associated with surfactant protein c dysfunction
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9857592/
https://www.ncbi.nlm.nih.gov/pubmed/36670712
http://dx.doi.org/10.3390/children10010162
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