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Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial

BACKGROUND: Systemic corticosteroid therapy failure is quite common in patients with newly diagnosed acute graft-versus-host disease (aGVHD). Growing evidence has suggested that mesenchymal stem cell (MSC) therapy could be a promising treatment option for aGVHD due to its distinctive immunomodulatin...

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Autores principales: Yang, Donglin, Hou, Xiaoqiang, Qian, Kun, Li, Yuhang, Hu, Liangding, Li, Liang, Han, Mingzhe, Yao, Chen, Liu, Daihong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10155385/
https://www.ncbi.nlm.nih.gov/pubmed/37138332
http://dx.doi.org/10.1186/s13063-023-07305-0
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author Yang, Donglin
Hou, Xiaoqiang
Qian, Kun
Li, Yuhang
Hu, Liangding
Li, Liang
Han, Mingzhe
Yao, Chen
Liu, Daihong
author_facet Yang, Donglin
Hou, Xiaoqiang
Qian, Kun
Li, Yuhang
Hu, Liangding
Li, Liang
Han, Mingzhe
Yao, Chen
Liu, Daihong
author_sort Yang, Donglin
collection PubMed
description BACKGROUND: Systemic corticosteroid therapy failure is quite common in patients with newly diagnosed acute graft-versus-host disease (aGVHD). Growing evidence has suggested that mesenchymal stem cell (MSC) therapy could be a promising treatment option for aGVHD due to its distinctive immunomodulating functions. However, there is a lack of randomized well-controlled clinical trials. METHODS: This is a clinical trial protocol for a multicenter, randomized, double-blind, placebo-controlled phase II study. The aim of the trial is to evaluate the efficacy and safety of the administration of the human umbilical cord-derived MSC product hUC-MSC PLEB001 in patients with grade II–IV, steroid-refractory aGVHD. A total of 96 patients will be randomized 1:1 to receive MSC or placebo treatment twice per week for 4 weeks, in addition to second-line therapy according to institutional standards. Patients who achieve partial response (PR) at day 28 will be eligible to receive further infusions twice per week for an additional 4 weeks. DISCUSSION: This study will evaluate the efficacy and safety of MSC therapy in patients who have failed first-line steroid treatment for grade II–IV aGVHD. TRIAL REGISTRATION: Chinese Clinical Trial Registry (ChiCTR), ChiCTR2000035740. Registered on 16 August 2020. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13063-023-07305-0.
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spelling pubmed-101553852023-05-04 Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial Yang, Donglin Hou, Xiaoqiang Qian, Kun Li, Yuhang Hu, Liangding Li, Liang Han, Mingzhe Yao, Chen Liu, Daihong Trials Study Protocol BACKGROUND: Systemic corticosteroid therapy failure is quite common in patients with newly diagnosed acute graft-versus-host disease (aGVHD). Growing evidence has suggested that mesenchymal stem cell (MSC) therapy could be a promising treatment option for aGVHD due to its distinctive immunomodulating functions. However, there is a lack of randomized well-controlled clinical trials. METHODS: This is a clinical trial protocol for a multicenter, randomized, double-blind, placebo-controlled phase II study. The aim of the trial is to evaluate the efficacy and safety of the administration of the human umbilical cord-derived MSC product hUC-MSC PLEB001 in patients with grade II–IV, steroid-refractory aGVHD. A total of 96 patients will be randomized 1:1 to receive MSC or placebo treatment twice per week for 4 weeks, in addition to second-line therapy according to institutional standards. Patients who achieve partial response (PR) at day 28 will be eligible to receive further infusions twice per week for an additional 4 weeks. DISCUSSION: This study will evaluate the efficacy and safety of MSC therapy in patients who have failed first-line steroid treatment for grade II–IV aGVHD. TRIAL REGISTRATION: Chinese Clinical Trial Registry (ChiCTR), ChiCTR2000035740. Registered on 16 August 2020. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13063-023-07305-0. BioMed Central 2023-05-03 /pmc/articles/PMC10155385/ /pubmed/37138332 http://dx.doi.org/10.1186/s13063-023-07305-0 Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Study Protocol
Yang, Donglin
Hou, Xiaoqiang
Qian, Kun
Li, Yuhang
Hu, Liangding
Li, Liang
Han, Mingzhe
Yao, Chen
Liu, Daihong
Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial
title Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial
title_full Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial
title_fullStr Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial
title_full_unstemmed Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial
title_short Efficacy and safety of human umbilical cord-derived mesenchymal stem cells (hUC-MSC PLEB001) for the treatment of grade II–IV steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase II trial
title_sort efficacy and safety of human umbilical cord-derived mesenchymal stem cells (huc-msc pleb001) for the treatment of grade ii–iv steroid-refractory acute graft-versus-host disease: a study protocol for a multicenter, randomized, double-blind, placebo-controlled, phase ii trial
topic Study Protocol
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10155385/
https://www.ncbi.nlm.nih.gov/pubmed/37138332
http://dx.doi.org/10.1186/s13063-023-07305-0
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