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The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report

BACKGROUND: Chronic granulomatous disease (CGD) is an inborn error of immunity (IEI) disorder that results from defects in the respiratory burst activity in phagocytes, leading to the inability to kill bacterial and fungal microorganisms. CGD patients usually have a high incidence of morbidity such...

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Autores principales: Nguyen-Thanh, Binh, Nguyen-Ngoc-Quynh, Le, Dang-Thi, Ha, Le-Quynh, Chi, Nguyen-Thi-Van, Anh, Thuc-Thanh, Huyen, Dang-Anh, Duong, Lee, Pamela P., Cao-Viet, Tung, Tran-Minh, Dien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10156979/
https://www.ncbi.nlm.nih.gov/pubmed/37153592
http://dx.doi.org/10.3389/fimmu.2023.1134852
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author Nguyen-Thanh, Binh
Nguyen-Ngoc-Quynh, Le
Dang-Thi, Ha
Le-Quynh, Chi
Nguyen-Thi-Van, Anh
Thuc-Thanh, Huyen
Dang-Anh, Duong
Lee, Pamela P.
Cao-Viet, Tung
Tran-Minh, Dien
author_facet Nguyen-Thanh, Binh
Nguyen-Ngoc-Quynh, Le
Dang-Thi, Ha
Le-Quynh, Chi
Nguyen-Thi-Van, Anh
Thuc-Thanh, Huyen
Dang-Anh, Duong
Lee, Pamela P.
Cao-Viet, Tung
Tran-Minh, Dien
author_sort Nguyen-Thanh, Binh
collection PubMed
description BACKGROUND: Chronic granulomatous disease (CGD) is an inborn error of immunity (IEI) disorder that results from defects in the respiratory burst activity in phagocytes, leading to the inability to kill bacterial and fungal microorganisms. CGD patients usually have a high incidence of morbidity such as infections and autoinflammatory diseases and a high mortality rate. Allogeneic bone marrow transplantation (BMT) is the only definitive cure for patients who suffer from CGD. CASE PRESENTATION: We report the first transplant case of chronic granulomatous disease in Vietnam. A 25-month-old boy with X-linked CGD underwent bone marrow transplantation from his 5-year-old, full-matched human leukocyte antigen (HLA)-carrier sibling after myeloablative conditioning regimen with busulfan 5.1 mg/kg/day for 4 days, fludarabine 30 mg/m(2)/day for 5 days, and rATG (Grafalon-Fresenius) 10 mg/kg/day for 4 days. Neutrophil was engrafted on day 13 posttransplant, donor chimerism was 100% on day 30 with the dihydrorhodamine-1,2,3 (DHR 123) flow cytometric assay test that reached 38% of the normal 45 days posttransplant. Five months after transplant, the patient was free of infection with stable DHR 123 assay at 37%, and donor chimerism remained 100%. No sign of a graft-versus-host disease had been observed posttransplant. CONCLUSION: We suggest that bone marrow transplantation is a safe and effectual cure for CGD patients, especially for patients with HLA-identical siblings.
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spelling pubmed-101569792023-05-05 The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report Nguyen-Thanh, Binh Nguyen-Ngoc-Quynh, Le Dang-Thi, Ha Le-Quynh, Chi Nguyen-Thi-Van, Anh Thuc-Thanh, Huyen Dang-Anh, Duong Lee, Pamela P. Cao-Viet, Tung Tran-Minh, Dien Front Immunol Immunology BACKGROUND: Chronic granulomatous disease (CGD) is an inborn error of immunity (IEI) disorder that results from defects in the respiratory burst activity in phagocytes, leading to the inability to kill bacterial and fungal microorganisms. CGD patients usually have a high incidence of morbidity such as infections and autoinflammatory diseases and a high mortality rate. Allogeneic bone marrow transplantation (BMT) is the only definitive cure for patients who suffer from CGD. CASE PRESENTATION: We report the first transplant case of chronic granulomatous disease in Vietnam. A 25-month-old boy with X-linked CGD underwent bone marrow transplantation from his 5-year-old, full-matched human leukocyte antigen (HLA)-carrier sibling after myeloablative conditioning regimen with busulfan 5.1 mg/kg/day for 4 days, fludarabine 30 mg/m(2)/day for 5 days, and rATG (Grafalon-Fresenius) 10 mg/kg/day for 4 days. Neutrophil was engrafted on day 13 posttransplant, donor chimerism was 100% on day 30 with the dihydrorhodamine-1,2,3 (DHR 123) flow cytometric assay test that reached 38% of the normal 45 days posttransplant. Five months after transplant, the patient was free of infection with stable DHR 123 assay at 37%, and donor chimerism remained 100%. No sign of a graft-versus-host disease had been observed posttransplant. CONCLUSION: We suggest that bone marrow transplantation is a safe and effectual cure for CGD patients, especially for patients with HLA-identical siblings. Frontiers Media S.A. 2023-04-20 /pmc/articles/PMC10156979/ /pubmed/37153592 http://dx.doi.org/10.3389/fimmu.2023.1134852 Text en Copyright © 2023 Nguyen-Thanh, Nguyen-Ngoc-Quynh, Dang-Thi, Le-Quynh, Nguyen-Thi-Van, Thuc-Thanh, Dang-Anh, Lee, Cao-Viet and Tran-Minh https://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 Immunology
Nguyen-Thanh, Binh
Nguyen-Ngoc-Quynh, Le
Dang-Thi, Ha
Le-Quynh, Chi
Nguyen-Thi-Van, Anh
Thuc-Thanh, Huyen
Dang-Anh, Duong
Lee, Pamela P.
Cao-Viet, Tung
Tran-Minh, Dien
The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report
title The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report
title_full The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report
title_fullStr The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report
title_full_unstemmed The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report
title_short The first successful bone marrow transplantation in Vietnam for a young Vietnamese boy with chronic granulomatous disease: a case report
title_sort first successful bone marrow transplantation in vietnam for a young vietnamese boy with chronic granulomatous disease: a case report
topic Immunology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10156979/
https://www.ncbi.nlm.nih.gov/pubmed/37153592
http://dx.doi.org/10.3389/fimmu.2023.1134852
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