阿伐曲波帕与标准免疫抑制疗法联合治疗重型再生障碍性贫血的有效性和安全性

IF 2.5 4区医学 Q2 HEMATOLOGY

Experimental hematology Pub Date : 2024-11-05 DOI:10.1016/j.exphem.2024.104670

Jianping Li , Weiru Liang , Huihui Fan , Kang Zhou , Yuan Li , Wenrui Yang , Liping Jing , Li Zhang , Lei Ye , Youzhen Xiong , Guangxin Peng , Yang Yang , Weiping Yuan , Jun Shi , Fengkui Zhang , Xin Zhao

{"title":"阿伐曲波帕与标准免疫抑制疗法联合治疗重型再生障碍性贫血的有效性和安全性","authors":"Jianping Li , Weiru Liang , Huihui Fan , Kang Zhou , Yuan Li , Wenrui Yang , Liping Jing , Li Zhang , Lei Ye , Youzhen Xiong , Guangxin Peng , Yang Yang , Weiping Yuan , Jun Shi , Fengkui Zhang , Xin Zhao","doi":"10.1016/j.exphem.2024.104670","DOIUrl":null,"url":null,"abstract":"<div><div>Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disease. The addition of eltrombopag to immunosuppressive therapy (IST) improves the response rate, but its hepatotoxicity is concerning. Avatrombopag (AVA), a small-molecule thrombopoietin-receptor agonist without hepatotoxicity, has unknown efficacy in SAA treatment. This retrospective study assessed the efficacy and safety of AVA added to IST 42 SAA patients compared to a historical cohort of 84 patients receiving IST alone, using propensity score matching. The median age was 31.5 (6.0–67.0 years) years old in group A and 26 (16.0–45.0 years) years old in group B. At 3 months, group A showed higher complete response (CR) and overall response (OR) rates than group B (CR: 19.0% vs. 4.8%, <em>p</em> = 0.024; OR: 54.8% vs. 39.3%, <em>p</em> = 0.145). Higher CR and OR rates were also found at 6 months in group A than in group B (CR: 31.0% vs. 14.3%, <em>p</em> = 0.145; OR 71.4% vs. 51.2%, <em>p</em> = 0.048). In multivariate analysis of group A, a shorter interval from disease onset to antithymocyte globulin (ATG) treatment (≤6 months) (<em>p</em> = 0.005) predicted better responses rate at 6 months. Event-free survival was also improved in group A (60.7% vs. 49.6%). AVA was well-tolerated, with no hepatic injury observed during treatment, even in those with pre-existing hepatic impairment. The addition of AVA to IST improves both the response rate and response quality in patients with SAA while ensuring safety.</div></div>","PeriodicalId":12202,"journal":{"name":"Experimental hematology","volume":"140 ","pages":"Article 104670"},"PeriodicalIF":2.5000,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Efficacy and safety of avatrombopag in combination with standard immunosuppressive therapy for severe aplastic anemia\",\"authors\":\"Jianping Li , Weiru Liang , Huihui Fan , Kang Zhou , Yuan Li , Wenrui Yang , Liping Jing , Li Zhang , Lei Ye , Youzhen Xiong , Guangxin Peng , Yang Yang , Weiping Yuan , Jun Shi , Fengkui Zhang , Xin Zhao\",\"doi\":\"10.1016/j.exphem.2024.104670\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disease. The addition of eltrombopag to immunosuppressive therapy (IST) improves the response rate, but its hepatotoxicity is concerning. Avatrombopag (AVA), a small-molecule thrombopoietin-receptor agonist without hepatotoxicity, has unknown efficacy in SAA treatment. This retrospective study assessed the efficacy and safety of AVA added to IST 42 SAA patients compared to a historical cohort of 84 patients receiving IST alone, using propensity score matching. The median age was 31.5 (6.0–67.0 years) years old in group A and 26 (16.0–45.0 years) years old in group B. At 3 months, group A showed higher complete response (CR) and overall response (OR) rates than group B (CR: 19.0% vs. 4.8%, <em>p</em> = 0.024; OR: 54.8% vs. 39.3%, <em>p</em> = 0.145). Higher CR and OR rates were also found at 6 months in group A than in group B (CR: 31.0% vs. 14.3%, <em>p</em> = 0.145; OR 71.4% vs. 51.2%, <em>p</em> = 0.048). In multivariate analysis of group A, a shorter interval from disease onset to antithymocyte globulin (ATG) treatment (≤6 months) (<em>p</em> = 0.005) predicted better responses rate at 6 months. Event-free survival was also improved in group A (60.7% vs. 49.6%). AVA was well-tolerated, with no hepatic injury observed during treatment, even in those with pre-existing hepatic impairment. The addition of AVA to IST improves both the response rate and response quality in patients with SAA while ensuring safety.</div></div>\",\"PeriodicalId\":12202,\"journal\":{\"name\":\"Experimental hematology\",\"volume\":\"140 \",\"pages\":\"Article 104670\"},\"PeriodicalIF\":2.5000,\"publicationDate\":\"2024-11-05\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Experimental hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0301472X24005356\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Experimental hematology","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0301472X24005356","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}

引用次数: 0

摘要

重型再生障碍性贫血（SAA）是一种危及生命的骨髓衰竭疾病。在免疫抑制疗法（IST）中加入艾曲波帕能提高再生障碍性贫血的应答率和应答质量，但其肝毒性令人担忧。阿瓦曲波帕（AVA）是另一种无肝脏毒性的小分子血小板生成素受体激动剂，但其在 SAA 治疗中的疗效尚不清楚。这项回顾性研究旨在评估小分子血小板生成素受体激动剂 AVA 加入 IST 治疗 SAA 患者的疗效和安全性。研究利用倾向评分匹配法，将接受 IST 和 AVA 治疗的 42 例患者与单独接受 IST 治疗的 84 例患者进行了比较。3 个月后，A 组的完全应答率（CR）和总应答率（OR）高于 B 组（CR：19.0% 对 4.8%，P=0.024；OR：54.8% 对 39.3%，P=0.145）。6 个月时，A 组的 CR 和 OR 率也高于 B 组（CR：31.0% 对 14.3%，P=0.145；OR：71.4% 对 51.2%，P=0.048）。在对A组的多变量分析中，从发病到接受抗胸腺细胞球蛋白（ATG）治疗的时间间隔越短（≤6个月）（P=0.005），6个月时的应答率就越高。A组的无事件生存率也有所提高（60.7% 对 49.6%）。AVA 的耐受性良好，在治疗过程中未观察到肝损伤，即使是那些原本就存在肝损伤的患者也是如此。在 IST 中加入 AVA 可提高 SAA 患者的应答率和应答质量，同时确保安全性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Efficacy and safety of avatrombopag in combination with standard immunosuppressive therapy for severe aplastic anemia

Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disease. The addition of eltrombopag to immunosuppressive therapy (IST) improves the response rate, but its hepatotoxicity is concerning. Avatrombopag (AVA), a small-molecule thrombopoietin-receptor agonist without hepatotoxicity, has unknown efficacy in SAA treatment. This retrospective study assessed the efficacy and safety of AVA added to IST 42 SAA patients compared to a historical cohort of 84 patients receiving IST alone, using propensity score matching. The median age was 31.5 (6.0–67.0 years) years old in group A and 26 (16.0–45.0 years) years old in group B. At 3 months, group A showed higher complete response (CR) and overall response (OR) rates than group B (CR: 19.0% vs. 4.8%, p = 0.024; OR: 54.8% vs. 39.3%, p = 0.145). Higher CR and OR rates were also found at 6 months in group A than in group B (CR: 31.0% vs. 14.3%, p = 0.145; OR 71.4% vs. 51.2%, p = 0.048). In multivariate analysis of group A, a shorter interval from disease onset to antithymocyte globulin (ATG) treatment (≤6 months) (p = 0.005) predicted better responses rate at 6 months. Event-free survival was also improved in group A (60.7% vs. 49.6%). AVA was well-tolerated, with no hepatic injury observed during treatment, even in those with pre-existing hepatic impairment. The addition of AVA to IST improves both the response rate and response quality in patients with SAA while ensuring safety.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Experimental hematology 医学-血液学

CiteScore

5.30

自引率

0.00%

发文量

审稿时长

58 days

期刊介绍： Experimental Hematology publishes new findings, methodologies, reviews and perspectives in all areas of hematology and immune cell formation on a monthly basis that may include Special Issues on particular topics of current interest. The overall goal is to report new insights into how normal blood cells are produced, how their production is normally regulated, mechanisms that contribute to hematological diseases and new approaches to their treatment. Specific topics may include relevant developmental and aging processes, stem cell biology, analyses of intrinsic and extrinsic regulatory mechanisms, in vitro behavior of primary cells, clonal tracking, molecular and omics analyses, metabolism, epigenetics, bioengineering approaches, studies in model organisms, novel clinical observations, transplantation biology and new therapeutic avenues.