Evidence map›Paper›PMID 42298304›Full record

Observational studyBritish journal of haematology2026

Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study.

Yuan Xu, Ying Wang, Ningling Wang, Liya He, Jing Ling, Mingwei Jin, Bingshou Xie, Shaohua Le, Meijuan Huang, Jie Peng and 20 more

Abstract readMulticenter StudyObservational Study
In one paragraph

Observational study in British journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Observational
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

30 authors.

Yuan XuState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.ORCID https://orcid.org/0009-0004-5333-7739
Ying WangShenzhen Children's Hospital, Shenzhen, China.
Ningling WangDepartment of Pediatrics, The Second Hospital of Anhui Medical University, Hefei, China.
Liya HeGuangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.ORCID https://orcid.org/0000-0002-7981-2786
Jing LingDepartment of Hematology, Children's Hospital of Soochow University, Suzhou, China.
Mingwei JinXuzhou Star Hospital, Xuzhou, China.
Bingshou XieDepartment of Hematology, Wenzhou People's Hospital, Wenzhou, China.
Shaohua LeDepartment of Pediatric Hematology, Fujian Medical University Union Hospital, Fuzhou, China.
Meijuan HuangDepartment of Hematology, Fujian Medical University Union Hospital, Fuzhou, China.
Jie PengDepartment of Haematology, Xiangya Hospital, Central South University, Changsha, Hunan, China.
Hongbo ChengDepartment of Hematology, Jiangxi Provincial People's Hospital, The First Affiliated Hospital of Nanchang Medical College, Nanchang, China.
Jiao JinDepartment of Pediatric Hematology, Affiliated Hospital of Guizhou Medical University, Guiyang, China.
Li ZhouDepartment of Hematology, The Second Hospital of Anhui Medical University, Hefei, China.ORCID https://orcid.org/0000-0002-3668-3558
Zeping ZhouDepartment of Hematology, The Second Affiliated Hospital of Kunming Medical University, Kunming, China.ORCID https://orcid.org/0000-0002-7698-7695
Can GaiDepartment of Hematology, Huaibei People's Hospital, Anhui Province, Huaibei, China.
Yujing ZhangDepartment of Hematology, The Fourth Hospital of Harbin Medical University, Harbin, Heilongjiang, China.
Mei SunQilu Hospital of Shandong University Dezhou Hospital, Dezhou, China.
Xiaojing LiChengdu New Century Women's and Children's Hospital, Chengdu, China.
Jie YinDepartment of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Yanli ChengDepartment of Hematology, Children's Hospital of Shanxi, Taiyuan, China.
Ailing XueDepartment of Pediatrics, Zibo Central Hospital, Shandong, Zibo, China.
Weiqun XuChildren's Hospital, Zhejiang University School of Medicine, Hangzhou, China.
Hong HanLiyuan Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.ORCID https://orcid.org/0000-0001-9691-7187
Fuhua ZhangDepartment of Hematology, Dongying People's Hospital, Dongying, Shandong Province, China.
Weiguo ZhuDepartment of Pediatrics, Foresea Life Insurance Shaoguan Hospital, Shaoguan, China.
Shen HeDepartment of Hematology, Kaifeng Central Hospital, Kaifeng, Henan, China.
Hua ZhouDepartment of Hematology and Oncology, Changzhou Children's Hospital, Changzhou, Jiangsu Province, China.
Lei ZhangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Feng XueState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.ORCID https://orcid.org/0000-0002-5947-3924
Renchi YangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.ORCID https://orcid.org/0000-0003-3741-8518

Funding

Chinese Academy of Medical Sciences (CAMS) Innovation Fund for Medical Sciences 2025-I2M-XHZY-034National Key Research and Development Program of China 2023YFC2507802National Natural Science Foundation of China 82470136National Natural Science Foundation of China 82470138Tianjin Municipal Science and Technology Commission 25ZXZSSS00510
6 · The paper itself

Abstract

Emicizumab is an established prophylactic therapy for haemophilia A (HA). However, real-world multicentre evidence on long-term outcomes, particularly in a resource-constrained setting, remains limited. We aimed to evaluate real-world outcomes in patients with HA receiving emicizumab prophylaxis across multiple centres with extended follow-up. We conducted a multicentre retrospective study of patients with HA who received emicizumab prophylaxis for ≥1 year across 25 centres. Outcomes included annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), bleeding events, target joint status and adverse events. A total of 132 patients (131 males and 1 female) were included, with a median age at switch of 2.0 years (interquartile range [IQR], 1.0-4.8). Over a median follow-up of 26 months (IQR: 16.0-40.0), the negative binomial regression model-based ABR for all bleeds was 0.81 (95% confidence interval [CI], 0.62-1.07). Across 12-month intervals, both calculated mean and negative binomial regression model-based AJBR remained between 0.1 and 0.3. Of the 74 baseline target joints, 95.9% (n = 71) resolved and no new target joints developed. Forty-two patients (31.8%) received factor concentrates during emicizumab treatment. Injection-site reactions occurred in 7.6% of patients, with no thromboembolic or fatal events observed. Long-term emicizumab prophylaxis provides sustained bleeding control, joint protection and a favourable safety profile.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedHemophilia AAdultChildChild, PreschoolChinaFemaleFollow-Up StudiesHemorrhageHumansInfantMaleRetrospective StudiesTreatment OutcomeAntibodies, BispecificAntibodies, Monoclonal, Humanizedemicizumabbleedingemicizumabhaemophilia Amachine learningprophylaxis

Identifiers

PMID42298304
PMCPMC13570176

What OpenQuestion holds

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Registered trials

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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.