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Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study

Thrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and life-threatening complication. Thus, new prevention and treatment strategies for post-HSCT thrombocytopenia are urgently required. In recent studies, thrombopoietin receptor agonists (TPO-RA) fo...

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Autores principales: Ruan, Yongsheng, Cao, Wei, Luo, Tingting, Liu, Xuan, Liu, Qiujun, Xiao, Yuhua, Wu, Cuiling, Xie, Danfeng, Ren, Yuqiong, Wu, Xuedong, Feng, Xiaoqin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9975496/
https://www.ncbi.nlm.nih.gov/pubmed/36873638
http://dx.doi.org/10.3389/fped.2023.1099372
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author Ruan, Yongsheng
Cao, Wei
Luo, Tingting
Liu, Xuan
Liu, Qiujun
Xiao, Yuhua
Wu, Cuiling
Xie, Danfeng
Ren, Yuqiong
Wu, Xuedong
Feng, Xiaoqin
author_facet Ruan, Yongsheng
Cao, Wei
Luo, Tingting
Liu, Xuan
Liu, Qiujun
Xiao, Yuhua
Wu, Cuiling
Xie, Danfeng
Ren, Yuqiong
Wu, Xuedong
Feng, Xiaoqin
author_sort Ruan, Yongsheng
collection PubMed
description Thrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and life-threatening complication. Thus, new prevention and treatment strategies for post-HSCT thrombocytopenia are urgently required. In recent studies, thrombopoietin receptor agonists (TPO-RA) for treating post-HSCT thrombocytopenia indicated efficiency and safety. The improved effect of post-HSCT thrombocytopenia in adults was found in the administration of avatrombopag which was a new TPO-RA. However, there was no relevant study in the children's cohort. Herein, we retrospectively analyzed the effect of avatrombopag in post-HSCT thrombocytopenia in children. As a result, the overall response rate (ORR) and complete response rate (CRR) were 91% and 78%, respectively. Furthermore, both cumulative ORR and CRR were significantly lower in the poor graft function (PGF)/secondary failure of platelet recovery (SFPR) group compared to the engraftment-promotion group (86.7% vs. 100%, p = 0.002 and 65.0% vs. 100%, p < 0.001, respectively). Achieving OR required a median of 16 days in the PGF/SFPR group while 7 days in the engraftment-promotion group (p = 0.003). Grade III–IV acute graft vs. host disease and inadequate megakaryocytes were identified as risk factors of CRR only in univariate analysis (p = 0.03 and p = 0.01, respectively). No severe adverse events were documented. Conclusively, avatrombopag is an alternatively efficient and safe agent for treating post-HSCT thrombocytopenia in children.
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spelling pubmed-99754962023-03-02 Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study Ruan, Yongsheng Cao, Wei Luo, Tingting Liu, Xuan Liu, Qiujun Xiao, Yuhua Wu, Cuiling Xie, Danfeng Ren, Yuqiong Wu, Xuedong Feng, Xiaoqin Front Pediatr Pediatrics Thrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and life-threatening complication. Thus, new prevention and treatment strategies for post-HSCT thrombocytopenia are urgently required. In recent studies, thrombopoietin receptor agonists (TPO-RA) for treating post-HSCT thrombocytopenia indicated efficiency and safety. The improved effect of post-HSCT thrombocytopenia in adults was found in the administration of avatrombopag which was a new TPO-RA. However, there was no relevant study in the children's cohort. Herein, we retrospectively analyzed the effect of avatrombopag in post-HSCT thrombocytopenia in children. As a result, the overall response rate (ORR) and complete response rate (CRR) were 91% and 78%, respectively. Furthermore, both cumulative ORR and CRR were significantly lower in the poor graft function (PGF)/secondary failure of platelet recovery (SFPR) group compared to the engraftment-promotion group (86.7% vs. 100%, p = 0.002 and 65.0% vs. 100%, p < 0.001, respectively). Achieving OR required a median of 16 days in the PGF/SFPR group while 7 days in the engraftment-promotion group (p = 0.003). Grade III–IV acute graft vs. host disease and inadequate megakaryocytes were identified as risk factors of CRR only in univariate analysis (p = 0.03 and p = 0.01, respectively). No severe adverse events were documented. Conclusively, avatrombopag is an alternatively efficient and safe agent for treating post-HSCT thrombocytopenia in children. Frontiers Media S.A. 2023-02-15 /pmc/articles/PMC9975496/ /pubmed/36873638 http://dx.doi.org/10.3389/fped.2023.1099372 Text en © 2023 Ruan, Cao, Luo, Liu, Liu, Xiao, Wu, Xie, Ren, Wu and Feng. 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) (https://creativecommons.org/licenses/by/4.0/) . 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
Ruan, Yongsheng
Cao, Wei
Luo, Tingting
Liu, Xuan
Liu, Qiujun
Xiao, Yuhua
Wu, Cuiling
Xie, Danfeng
Ren, Yuqiong
Wu, Xuedong
Feng, Xiaoqin
Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
title Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
title_full Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
title_fullStr Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
title_full_unstemmed Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
title_short Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
title_sort avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: a pilot study
topic Pediatrics
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9975496/
https://www.ncbi.nlm.nih.gov/pubmed/36873638
http://dx.doi.org/10.3389/fped.2023.1099372
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