Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective measure for the treatment of hematological disease. With the progress and wide use of allo-HSCT, post-transplant central nervous system complications (CNSC) have gotten more and more attention because of its poor prognosi...

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Main Authors: Ruirui Gui, Zhen Li, Juan Wang, Yingling Zu, Binglei Zhang, Juanjuan Zhao, Yongping Song, Jian Zhou
Format: Article
Language:English
Published: Elsevier 2024-11-01
Series:Heliyon
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Online Access:http://www.sciencedirect.com/science/article/pii/S2405844024163876
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author Ruirui Gui
Zhen Li
Juan Wang
Yingling Zu
Binglei Zhang
Juanjuan Zhao
Yongping Song
Jian Zhou
author_facet Ruirui Gui
Zhen Li
Juan Wang
Yingling Zu
Binglei Zhang
Juanjuan Zhao
Yongping Song
Jian Zhou
author_sort Ruirui Gui
collection DOAJ
description Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective measure for the treatment of hematological disease. With the progress and wide use of allo-HSCT, post-transplant central nervous system complications (CNSC) have gotten more and more attention because of its poor prognosis and high mortality. Since there is no standard treatment for patients with immune-mediated CNSC currently, new treatments are needed to be developed urgently. Here, we attempted a novel therapy regimen of intrathecal tocilizumab injection in two pediatric patients with immune-mediated CNSC manifested as cytokine release syndrome (CRS) after haploidentical hematopoietic stem cell transplantation (halpo-HSCT). In the two patients, persistent seizure symptoms could not be resolved 7 h after intravenous tocilizumab, while the symptoms were controlled rapidly only 2 hours or 1 hour after the first intrathecal injection of tocilizumab. Moreover, the level of interleukin 6 in the cerebrospinal fluid returned to normal after the fifth intrathecal injection. Even more appealing, no acute or chronic adverse reactions were observed during injection and subsequent follow-up. In conclusion, intrathecal tocilizumab seems to be more rapid and effective than intravenous administration for immune-mediated CNSC manifested as CRS in haplo-HSCT recipients. We recommend this treatment modality for further investigation.
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issn 2405-8440
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spelling doaj-art-3c193627567d4357958c6642b5f3b8542024-11-30T07:12:39ZengElsevierHeliyon2405-84402024-11-011022e40356Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reportsRuirui Gui0Zhen Li1Juan Wang2Yingling Zu3Binglei Zhang4Juanjuan Zhao5Yongping Song6Jian Zhou7Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, ChinaDepartment of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, ChinaDepartment of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, ChinaDepartment of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, ChinaDepartment of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, ChinaDepartment of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, ChinaDepartment of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, ChinaDepartment of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, China; Corresponding author. Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, 450000, China.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective measure for the treatment of hematological disease. With the progress and wide use of allo-HSCT, post-transplant central nervous system complications (CNSC) have gotten more and more attention because of its poor prognosis and high mortality. Since there is no standard treatment for patients with immune-mediated CNSC currently, new treatments are needed to be developed urgently. Here, we attempted a novel therapy regimen of intrathecal tocilizumab injection in two pediatric patients with immune-mediated CNSC manifested as cytokine release syndrome (CRS) after haploidentical hematopoietic stem cell transplantation (halpo-HSCT). In the two patients, persistent seizure symptoms could not be resolved 7 h after intravenous tocilizumab, while the symptoms were controlled rapidly only 2 hours or 1 hour after the first intrathecal injection of tocilizumab. Moreover, the level of interleukin 6 in the cerebrospinal fluid returned to normal after the fifth intrathecal injection. Even more appealing, no acute or chronic adverse reactions were observed during injection and subsequent follow-up. In conclusion, intrathecal tocilizumab seems to be more rapid and effective than intravenous administration for immune-mediated CNSC manifested as CRS in haplo-HSCT recipients. We recommend this treatment modality for further investigation.http://www.sciencedirect.com/science/article/pii/S2405844024163876TocilizumabImmune-mediated central nervous system complicationHaploidentical hematopoietic stem cell transplantationIntrathecal therapyCase report
spellingShingle Ruirui Gui
Zhen Li
Juan Wang
Yingling Zu
Binglei Zhang
Juanjuan Zhao
Yongping Song
Jian Zhou
Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports
Heliyon
Tocilizumab
Immune-mediated central nervous system complication
Haploidentical hematopoietic stem cell transplantation
Intrathecal therapy
Case report
title Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports
title_full Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports
title_fullStr Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports
title_full_unstemmed Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports
title_short Intrathecal tocilizumab for immune-mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children: Two case reports
title_sort intrathecal tocilizumab for immune mediated central nervous system complication after haploidentical hematopoietic stem cell transplantation in children two case reports
topic Tocilizumab
Immune-mediated central nervous system complication
Haploidentical hematopoietic stem cell transplantation
Intrathecal therapy
Case report
url http://www.sciencedirect.com/science/article/pii/S2405844024163876
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