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Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases

Aim: Anoxic brain injury (ABI) due to non-fatal drowning may cause persistent vegetative state (VS) that is currently incurable. The aim of this paper is to present the safety and feasibility of autologous bone marrow-derived mononuclear cell (BMMNC) transplantation in five drowning children survivi...

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Autores principales: Liem, Nguyen Thanh, Chinh, Vu Duy, Phuong, Dam Thi Minh, Van Doan, Ngo, Forsyth, Nicholas R., Heke, Michael, Thi, Phuong Anh Nguyen, Nguyen, Xuan-Hung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7511512/
https://www.ncbi.nlm.nih.gov/pubmed/33014944
http://dx.doi.org/10.3389/fped.2020.00564
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author Liem, Nguyen Thanh
Chinh, Vu Duy
Phuong, Dam Thi Minh
Van Doan, Ngo
Forsyth, Nicholas R.
Heke, Michael
Thi, Phuong Anh Nguyen
Nguyen, Xuan-Hung
author_facet Liem, Nguyen Thanh
Chinh, Vu Duy
Phuong, Dam Thi Minh
Van Doan, Ngo
Forsyth, Nicholas R.
Heke, Michael
Thi, Phuong Anh Nguyen
Nguyen, Xuan-Hung
author_sort Liem, Nguyen Thanh
collection PubMed
description Aim: Anoxic brain injury (ABI) due to non-fatal drowning may cause persistent vegetative state (VS) that is currently incurable. The aim of this paper is to present the safety and feasibility of autologous bone marrow-derived mononuclear cell (BMMNC) transplantation in five drowning children surviving in persistent VS. Methods: We used BMMNC as a novel candidate therapeutic tool in a pilot phase-I study for five patients affected by neurological sequelae after near-death drowning. Autologous BMMNCs were freshly isolated using Ficoll gradient centrifugation then infused intrathecally to five patients. The number of transplantation varied from two to four times depending on the motor function improvement of patient after transplantation. Clinical therapeutic effects were evaluated using gross motor function measure and muscle spasticity rating scales, cognitive assessments, and brain MRI before and after cell administrations. Results: Six months after BMMNC transplantation, no serious complications or adverse events were reported. All five patients displayed improvement across the major parameters of gross motor function, cognition, and muscle spasticity. Three patients displayed improved communication including the expression of words. In particular, one patient remarkably reduced cerebral atrophy, with nearly normal cerebral parenchyma after BMMNC transplantation. Conclusions: Autologous BMMNC transplantation for the treatment of children in persistent VS after drowning is safe, feasible, and can potentially improve motor function and cognition and reduce muscle spasticity. These results pave the way for a future phase II clinical trial to evaluate the efficacy of the therapy.
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spelling pubmed-75115122020-10-02 Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases Liem, Nguyen Thanh Chinh, Vu Duy Phuong, Dam Thi Minh Van Doan, Ngo Forsyth, Nicholas R. Heke, Michael Thi, Phuong Anh Nguyen Nguyen, Xuan-Hung Front Pediatr Pediatrics Aim: Anoxic brain injury (ABI) due to non-fatal drowning may cause persistent vegetative state (VS) that is currently incurable. The aim of this paper is to present the safety and feasibility of autologous bone marrow-derived mononuclear cell (BMMNC) transplantation in five drowning children surviving in persistent VS. Methods: We used BMMNC as a novel candidate therapeutic tool in a pilot phase-I study for five patients affected by neurological sequelae after near-death drowning. Autologous BMMNCs were freshly isolated using Ficoll gradient centrifugation then infused intrathecally to five patients. The number of transplantation varied from two to four times depending on the motor function improvement of patient after transplantation. Clinical therapeutic effects were evaluated using gross motor function measure and muscle spasticity rating scales, cognitive assessments, and brain MRI before and after cell administrations. Results: Six months after BMMNC transplantation, no serious complications or adverse events were reported. All five patients displayed improvement across the major parameters of gross motor function, cognition, and muscle spasticity. Three patients displayed improved communication including the expression of words. In particular, one patient remarkably reduced cerebral atrophy, with nearly normal cerebral parenchyma after BMMNC transplantation. Conclusions: Autologous BMMNC transplantation for the treatment of children in persistent VS after drowning is safe, feasible, and can potentially improve motor function and cognition and reduce muscle spasticity. These results pave the way for a future phase II clinical trial to evaluate the efficacy of the therapy. Frontiers Media S.A. 2020-09-10 /pmc/articles/PMC7511512/ /pubmed/33014944 http://dx.doi.org/10.3389/fped.2020.00564 Text en Copyright © 2020 Liem, Chinh, Phuong, Van Doan, Forsyth, Heke, Thi and Nguyen. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Pediatrics
Liem, Nguyen Thanh
Chinh, Vu Duy
Phuong, Dam Thi Minh
Van Doan, Ngo
Forsyth, Nicholas R.
Heke, Michael
Thi, Phuong Anh Nguyen
Nguyen, Xuan-Hung
Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases
title Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases
title_full Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases
title_fullStr Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases
title_full_unstemmed Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases
title_short Outcomes of Bone Marrow-Derived Mononuclear Cell Transplantation for Patients in Persistent Vegetative State After Drowning: Report of Five Cases
title_sort outcomes of bone marrow-derived mononuclear cell transplantation for patients in persistent vegetative state after drowning: report of five cases
topic Pediatrics
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7511512/
https://www.ncbi.nlm.nih.gov/pubmed/33014944
http://dx.doi.org/10.3389/fped.2020.00564
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