Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China

Background: Eltrombopag (ELT), a thrombopoietin receptor agonist (TPO-RA), has been approved for relapsed/refractory aplastic anemia (AA). However, data on avatrombopag (AVA), another TPO-RA, are limited, and the comparisons between the two TPO-RAs are lacking. Objectives: We aimed to compare the ef...

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Main Authors: Zhuxin Zhang, Qinglin Hu, Chen Yang, Miao Chen, Bing Han
Format: Article
Language:English
Published: SAGE Publishing 2023-09-01
Series:Therapeutic Advances in Hematology
Online Access:https://doi.org/10.1177/20406207231191310
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author Zhuxin Zhang
Qinglin Hu
Chen Yang
Miao Chen
Bing Han
author_facet Zhuxin Zhang
Qinglin Hu
Chen Yang
Miao Chen
Bing Han
author_sort Zhuxin Zhang
collection DOAJ
description Background: Eltrombopag (ELT), a thrombopoietin receptor agonist (TPO-RA), has been approved for relapsed/refractory aplastic anemia (AA). However, data on avatrombopag (AVA), another TPO-RA, are limited, and the comparisons between the two TPO-RAs are lacking. Objectives: We aimed to compare the efficacy and safety between ELT and AVA in relapsed/refractory AA patients. Design: In this retrospective study, patients with relapsed/refractory AA who had been treated with ELT ( N  = 45) or AVA ( N  = 30) alone and had compatible baseline hematological parameters were compared. Methods: Data from patients diagnosed with acquired AA were retrospectively collected. All patients were refractory/relapsed to standard immunosuppressive therapy (IST) for at least 6 months before ELT or AVA. Patients had to be treated with ELT or AVA alone for at least 6 months before evaluation if they did not respond. Baseline characteristics, overall response (OR), complete response (CR), relapse, adverse events, and factors that may affect efficacy were analyzed. Results: Of the 75 patients enrolled, 45 received ELT and 30 received AVA. Patients with AVA had a higher percentage of abnormal liver or renal function than those with ELT ( p =  0.036). No significant difference was found in the OR/CR rate in the first/second/third/sixth month between the two cohorts ( p >  0.05). Patients treated with AVA had a shorter median time to response than those treated with ELT ( p =  0.012) and had a higher platelet level in the second month ( p =  0.041). AVA had fewer adverse events than ELT ( p =  0.046). Under compatible follow-up time ( p =  0.463), no difference was found between the ELT and AVA cohorts in relapse ( p =  1.000) or clone evolution ( p =  0.637). No predictive factors for OR and CR in the sixth month were found for either ELT or AVA. Conclusion: With worse liver or renal function, AVA had a similar OR/CR rate but a shorter median time to response and fewer adverse events for patients with relapsed/refractory AA.
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spelling doaj.art-a0740bba112f42d8b51ccf767f6a5d0d2023-09-15T01:03:34ZengSAGE PublishingTherapeutic Advances in Hematology2040-62152023-09-011410.1177/20406207231191310Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in ChinaZhuxin ZhangQinglin HuChen YangMiao ChenBing HanBackground: Eltrombopag (ELT), a thrombopoietin receptor agonist (TPO-RA), has been approved for relapsed/refractory aplastic anemia (AA). However, data on avatrombopag (AVA), another TPO-RA, are limited, and the comparisons between the two TPO-RAs are lacking. Objectives: We aimed to compare the efficacy and safety between ELT and AVA in relapsed/refractory AA patients. Design: In this retrospective study, patients with relapsed/refractory AA who had been treated with ELT ( N  = 45) or AVA ( N  = 30) alone and had compatible baseline hematological parameters were compared. Methods: Data from patients diagnosed with acquired AA were retrospectively collected. All patients were refractory/relapsed to standard immunosuppressive therapy (IST) for at least 6 months before ELT or AVA. Patients had to be treated with ELT or AVA alone for at least 6 months before evaluation if they did not respond. Baseline characteristics, overall response (OR), complete response (CR), relapse, adverse events, and factors that may affect efficacy were analyzed. Results: Of the 75 patients enrolled, 45 received ELT and 30 received AVA. Patients with AVA had a higher percentage of abnormal liver or renal function than those with ELT ( p =  0.036). No significant difference was found in the OR/CR rate in the first/second/third/sixth month between the two cohorts ( p >  0.05). Patients treated with AVA had a shorter median time to response than those treated with ELT ( p =  0.012) and had a higher platelet level in the second month ( p =  0.041). AVA had fewer adverse events than ELT ( p =  0.046). Under compatible follow-up time ( p =  0.463), no difference was found between the ELT and AVA cohorts in relapse ( p =  1.000) or clone evolution ( p =  0.637). No predictive factors for OR and CR in the sixth month were found for either ELT or AVA. Conclusion: With worse liver or renal function, AVA had a similar OR/CR rate but a shorter median time to response and fewer adverse events for patients with relapsed/refractory AA.https://doi.org/10.1177/20406207231191310
spellingShingle Zhuxin Zhang
Qinglin Hu
Chen Yang
Miao Chen
Bing Han
Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China
Therapeutic Advances in Hematology
title Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China
title_full Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China
title_fullStr Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China
title_full_unstemmed Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China
title_short Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China
title_sort comparison of eltrombopag and avatrombopag in the treatment of refractory relapsed aplastic anemia a single center retrospective study in china
url https://doi.org/10.1177/20406207231191310
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