Evidence map›Paper›PMID 39088061›Full record

ArticleAnnals of hematology2024

Comparison of efficacy of eltrombopag combined with immunosuppression in the treatment of severe aplastic anemia and very severe aplastic anemia: real-world data and evidence.

Xiaoli Li, Xiaohui Shangguan, Hong Wang, Qingyuan Wang, Yanming Zhang, Bing Han, Rongrong Liu, Fengkui Zhang, Rong Fu, Zenghua Lin and 5 more

Abstract readComparative StudyMulticenter Study
PubMed Publisher
In one paragraph

Article in Annals of hematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed.

  1. Trial
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Xiaoli Li *Soochow Hopes Hematonosis Hospital, Suzhou, Jiangsu, China.
Xiaohui Shangguan *Department of Hematology, Longyan First Hospital, Affiliated to Fujian Medical University, Longyan, Fujian, China.
Hong WangNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Suzhou, Jiangsu, China.
Qingyuan WangNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Suzhou, Jiangsu, China.
Yanming ZhangDepartment of Hematology, The Affiliated Huai'an Hospital of Xuzhou Medical University and The Second People's Hospital of Huai'an, Huai'an, Jiangsu, China.
Bing HanDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Rongrong LiuDepartment of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China.
Fengkui ZhangState Key Laboratory of Experimental Hematology, Anemia Therapeutic Center, Institute of Hematology & Blood Diseases Hospital, National Clinical Research Center for Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Rong FuDepartment of Hematology, General Hospital, Tianjin Medical University, Tianjin, China.
Zenghua LinDepartment of Hematology, The Affiliated Hospital of Nantong University, Nantong, Jiangsu, China.
Miao MiaoNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Suzhou, Jiangsu, China.
Xiao MaNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Suzhou, Jiangsu, China.
Meiqing LeiDepartment of Hematology in Haikou Municipal People's Hospital, Affiliated Haikou Hospital Xiangya School of Medicine Central South University, Haikou, Hainan, China.
Depei WuNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Suzhou, Jiangsu, China. wudepei@suda.edu.cn.
Limin LiuNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Suzhou, Jiangsu, China. liminliu2006@163.com.

Funding

Innovation Capability Development Project of Jiangsu Province BM2015004Jiangsu Medical Outstanding Talents Project JCRCA2016002Jiangsu Provincial Key Medical Center YXZXA2016002National Key Research and Development Program of China 2016YFC0902800National Key Research and Development Program of China 2017YFA0104502National Key Research and Development Program of China 2017ZX09304021Priority Academic Program Development of Jiangsu Higher Education Institutions (PAPD) and the Science Foundation of Suzhou No.SKY2021040
6 · The paper itself

Abstract

Eltrombopag combined with immunosuppressive therapy (IST) was superior to IST alone for severe aplastic anemia (SAA) in the previous studies. But in China, horse antithymocyte globulin (hATG) is not available, instead, we use rabbit ATG (rATG). Here, we compared the efficacy and safety of IST (rATG combined with cyclosporine) combined with or without eltrombopag for the first-line treatment of SAA and very severe aplastic anemia (VSAA). A total of 371 patients in ten institutions in China from April 1, 2017 to December 1, 2022 were enrolled. The overall response (OR) rate at 3 months (54.2% vs. 41%; P = 0.046), the complete response (CR) (31.3% vs. 19.4%; P = 0.041) and OR (78.3% vs. 51.1%; P < 0.0001) rates at 6 months were significantly higher with IST combined with eltrombopag than with IST alone in SAA patients. While in VSAA patients, the addition of eltrombopag to IST only increased the CR rate at 6 months (29.8% vs. 9.43%; P = 0.010). Liver injury increased significantly in groups treated with IST combined with eltrombopag (P < 0.05). Serious treatment-related toxicities were similar (P > 0.05). In patients with SAA, 3-year failure-free survival (FFS) of eltrombopag combined with IST group was significantly higher than that of IST group (70.7 ± 5.3% vs. 50.3 ± 3.9%; P = 0.007). In patients with VSAA, the addition of eltrombopag significantly improved 3-year overall survival (OS) (82.2 ± 5.7% vs. 57.3 ± 7.2%; P = 0.020). Our findings suggested that IST combined with eltrombopag could improve the hematological recovery of newly diagnosed SAA without increasing severe toxicities. But in VSAA, the addition of eltrombopag seemed to show no other improvement to efficacy except the CR rate at 6 months.

Indexed as

Anemia, AplasticAntilymphocyte SerumBenzoatesHydrazinesImmunosuppressive AgentsPyrazolesAdolescentAdultAgedChildChild, PreschoolChinaCyclosporineDrug Therapy, CombinationFemaleHumansAntilymphocyte SerumBenzoatesCyclosporineeltrombopagHydrazinesImmunosuppressive AgentsPyrazolesEltrombopagImmunosuppressionSevere aplastic anemiaTreatmentVery severe aplastic anemia

Identifiers

PMID39088061

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.