Efficacy, Safety, and Population Pharmacokinetics of Eltrombopag in Children with Different Severities of Aplastic Anemia
J Clin Pharmacol. 2024 Mar 18. doi: 10.1002/jcph.2430. Online ahead of print.ABSTRACTEltrombopag was approved as a first-line treatment for patients older than 2 years old with severe aplastic anemia (SAA). However, data on eltrombopag in children with different types of aplastic anemia (AA), especially non-severe AA (NSAA), are limited. We performed a prospective, single-arm, and observational study to investigate eltrombopag's efficacy, safety, and pharmacokinetics in children with NSAA, SAA, and very severe AA (VSAA). The efficacy and safety were assessed every 3 months. The population pharmacokinetic (PPK) model was us...
Source: The Journal of Clinical Pharmacology - March 18, 2024 Category: Drugs & Pharmacology Authors: Wei Zhang Li-Xian Chang Bei-Bei Zhao Yi Zheng Dan-Dan Shan Bo-Hao Tang Fan Yang Yue Zhou Guo-Xiang Hao Ya-Hui Zhang van den Anker John Xiao-Fan Zhu Li Zhang Wei Zhao Source Type: research

Novel Perspectives on Thrombopoietin Receptor Agonists Applications
Hamostaseologie. 2024 Mar 18. doi: 10.1055/a-2250-9369. Online ahead of print.ABSTRACTSecond-generation thrombopoietin receptor agonists (TPO-RAs), romiplostim, eltrombopag, and avatrombopag, have been proved to be significant stimulators of megakaryopoiesis and, in the last decade, they have been incorporated in the treatment options against refractory immune thrombocytopenia in children and adults that do not respond to conventional therapy. Additionally, given their beneficial impact on hematopoiesis, they have successfully been applied in cases of non-immune thrombocytopenia, such as aplastic anemia, HCV-related thromb...
Source: Hamostaseologie - March 18, 2024 Category: Hematology Authors: Christos Stafylidis Dimitra Vlachopoulou Stavroula Syriopoulou Sevastianos Chatzidavid Nora-Athina Viniou Source Type: research

Efficacy, Safety, and Population Pharmacokinetics of Eltrombopag in Children with Different Severities of Aplastic Anemia
J Clin Pharmacol. 2024 Mar 18. doi: 10.1002/jcph.2430. Online ahead of print.ABSTRACTEltrombopag was approved as a first-line treatment for patients older than 2 years old with severe aplastic anemia (SAA). However, data on eltrombopag in children with different types of aplastic anemia (AA), especially non-severe AA (NSAA), are limited. We performed a prospective, single-arm, and observational study to investigate eltrombopag's efficacy, safety, and pharmacokinetics in children with NSAA, SAA, and very severe AA (VSAA). The efficacy and safety were assessed every 3 months. The population pharmacokinetic (PPK) model was us...
Source: The Journal of Clinical Pharmacology - March 18, 2024 Category: Drugs & Pharmacology Authors: Wei Zhang Li-Xian Chang Bei-Bei Zhao Yi Zheng Dan-Dan Shan Bo-Hao Tang Fan Yang Yue Zhou Guo-Xiang Hao Ya-Hui Zhang van den Anker John Xiao-Fan Zhu Li Zhang Wei Zhao Source Type: research

Novel Perspectives on Thrombopoietin Receptor Agonists Applications
Hamostaseologie. 2024 Mar 18. doi: 10.1055/a-2250-9369. Online ahead of print.ABSTRACTSecond-generation thrombopoietin receptor agonists (TPO-RAs), romiplostim, eltrombopag, and avatrombopag, have been proved to be significant stimulators of megakaryopoiesis and, in the last decade, they have been incorporated in the treatment options against refractory immune thrombocytopenia in children and adults that do not respond to conventional therapy. Additionally, given their beneficial impact on hematopoiesis, they have successfully been applied in cases of non-immune thrombocytopenia, such as aplastic anemia, HCV-related thromb...
Source: Hamostaseologie - March 18, 2024 Category: Hematology Authors: Christos Stafylidis Dimitra Vlachopoulou Stavroula Syriopoulou Sevastianos Chatzidavid Nora-Athina Viniou Source Type: research

Comparable outcomes with low-dose and standard-dose horse anti-thymocyte globulin in the treatment of severe aplastic anemia
CONCLUSION: Our study revealed that the response rates of patients with AA and LD were similar to those of patients with SD to hATG combined with cyclosporine in a real-world setting.PMID:38485832 | DOI:10.1007/s44313-024-00003-z (Source: Blood Research)
Source: Blood Research - March 15, 2024 Category: Hematology Authors: Arihant Jain Aditya Jandial Thenmozhi Mani Kamal Kishore Charanpreet Singh Deepesh Lad Gaurav Prakash Alka Khadwal Reena Das Neelam Varma Subhash Varma Pankaj Malhotra Source Type: research

Comparable outcomes with low-dose and standard-dose horse anti-thymocyte globulin in the treatment of severe aplastic anemia
CONCLUSION: Our study revealed that the response rates of patients with AA and LD were similar to those of patients with SD to hATG combined with cyclosporine in a real-world setting.PMID:38485832 | PMC:PMC10903521 | DOI:10.1007/s44313-024-00003-z (Source: Blood Research)
Source: Blood Research - March 15, 2024 Category: Hematology Authors: Arihant Jain Aditya Jandial Thenmozhi Mani Kamal Kishore Charanpreet Singh Deepesh Lad Gaurav Prakash Alka Khadwal Reena Das Neelam Varma Subhash Varma Pankaj Malhotra Source Type: research

Unraveling trajectories from aplastic anemia to hematologic malignancies: genetic and molecular insights
ConclusionThis study highlights the genetic dynamics in the progression from AA to hematologic malignancy. Monosomy 7’s prevalence and the occurrence of PTPN11 mutations suggest predictive and prognostic significance. Clonal evolution underscores the complexity of disease progression. (Source: Frontiers in Oncology)
Source: Frontiers in Oncology - March 13, 2024 Category: Cancer & Oncology Source Type: research

Dioscin alleviates aplastic anemia through regulatory T cells promotion
Conclusion: Dioscin markedly attenuated bone marrow failure, and promoted Tregs differentiation, suggesting the maintenance of theimmune balance effect of Dioscin. Dioscin attenuates pancytopenia and bone marrow failure via its Tregs promotion properties.PMID:38466633 | DOI:10.1080/16078454.2024.2326389 (Source: Hematology)
Source: Hematology - March 11, 2024 Category: Hematology Authors: Liwei Fan Runfeng Ni Haijin Wang Le Zhang Aidi Wang Baoshan Liu Source Type: research

HERVs may  perform as the initial trigger for acquired aplastic anemia
J Transl Med. 2024 Mar 9;22(1):260. doi: 10.1186/s12967-024-05052-7.NO ABSTRACTPMID:38461345 | DOI:10.1186/s12967-024-05052-7 (Source: Cancer Control)
Source: Cancer Control - March 9, 2024 Category: Cancer & Oncology Authors: Ting Wang Nianbin Li Heng Wu Rong Fu Source Type: research