ArticleFrontiers in pediatrics2022
Real-world experience of emicizumab prophylaxis in young children with hemophilia A: retrospective data from China.
Article in Frontiers in pediatrics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
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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.
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.
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 32 citations in OpenAlex.
- Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis.Haemophilia : the official journal of the World Federation of HemophiliaPooled it
- [Evaluation of the efficacy and safety of reduced-dose emicizumab in infants with severe hemophilia A: a single-center real-world study].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026Article
- Emicizumab prophylaxis beyond clinical trials: a multicenter, prospective real-world study of pediatric hemophilia patients with and without inhibitors.European journal of pediatrics · 2026Article
- Twelve-month follow-up of a real-world, noninterventional study evaluating the impact of emicizumab on bleeding episodes, joint health, and quality of life in people with hemophilia A.Therapeutic advances in hematology · 2026Article
- Emicizumab in Children with Severe Hemophilia A.Indian journal of pediatrics · 2025Observational
- Emicizumab utilization, safety, and outcomes in people with severe hemophilia and no inhibitors: 3-year follow-up. A report from the UK Haemophilia Centre Doctors' Organisation.Research and practice in thrombosis and haemostasis · 2025Article
- Real-world bleeding rates on emicizumab: the value of using nationwide digital treatment diary data in clinical research.Research and practice in thrombosis and haemostasis · 2025Article
- Importance of Inhibitor Surveillance During Emicizumab Prophylaxis in Young Children With Hemophilia: An Illustrative Case Series.Case reports in hematology · 2025Article
- Contemporary approaches to treat people with hemophilia: what's new and what's not?Research and practice in thrombosis and haemostasis · 2025Review
- A Prospective, Noninterventional Study to Evaluate the Impact of Emicizumab in the Management of Hemophilia A.Cureus · 2024Article
- Real-world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center.Pediatric investigation · 2024Article
- Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review.Research and practice in thrombosis and haemostasis · 2024Article
- Review
- Emicizumab prophylaxis for people with hemophilia A: Waste estimation and the Brazilian perspective.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
- [Chinese guidelines on the diagnosis and management of hemophilia with inhibitors (2023)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2023Article
- Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis.Research and practice in thrombosis and haemostasis · 2023Article
- Post‑Marketing Surveillance of the Safety and Effectiveness of Emicizumab in Japanese Patients With Congenital Haemophilia A With Inhibitors.Haemophilia : the official journal of the World Federation of HemophiliaArticle
- Multicentre Retrospective and Prospective Analysis of Data on Emicizumab and Factor VIII Prophylaxis in Children With Severe Haemophilia A: Egypt-Compare Study.Haemophilia : the official journal of the World Federation of HemophiliaArticle
- Lifetime Cost-Utility Analysis of Emicizumab Prophylaxis in Severe Haemophilia A Without Inhibitors in China: A Markov Model Analysis.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: As a new non-factor therapy for hemophilia A (HA), real-world study of emicizumab is still scarce. This study aimed to investigate the real-world use of emicizumab in Chinese boys with HA. Methods: Patients with moderate or severe HA were enrolled at Beijing Children's Hospital. They take emicizumab weekly (3 mg/kg) for a month and then went into a maintenance period with a different dosing regimen. After obtaining platelet-poor plasma at end of the loading period and during the maintenance period, coagulation ability and FVIII inhibitor were determined using human and bovine chromogenic Bethesda assay. Patients' bleeding rates were calculated through patients' records from 24 weeks before to at least 6 months after the switch (to emicizumab). Result: In total, 13 pediatric patients with HA (severe: moderate = 11:2) were enrolled in this study. The patients' age was 3.51 (0.73-6.65) years. Eight had FVIII inhibitors at enrollment and one of them developed FVIII inhibitors again during the switch. The coagulation level of the maintenance period was 19.6 (13.5-32.8) IU/dL ( Conclusion: Emicizumab could reduce bleeds in pediatric patients with/without FVIII inhibitors and eliminate target joints.
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Registered trials
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.