ArticleBMC pediatrics2022
Emicizumab in children: bleeding episodes and outcome before and after transition to Emicizumab.
Article in BMC pediatrics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 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.
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Who cites it
8 citing papers in PubMed, 1 synthesis or guideline pooled it, 16 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
- Emicizumab prophylaxis beyond clinical trials: a multicenter, prospective real-world study of pediatric hemophilia patients with and without inhibitors.European journal of pediatrics · 2026Article
- Impact of Family History of Haemophilia on Diagnosis, Management and Outcomes in Severe Haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 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
- Contemporary approaches to treat people with hemophilia: what's new and what's not?Research and practice in thrombosis and haemostasis · 2025Review
- Emicizumab: the hemophilia A game-changer.Haematologica · 2024Review
- 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
- Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis.Research and practice in thrombosis and haemostasis · 2023Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeReal-world data and study data regarding therapy with Emicizumab in pediatric cohorts with haemophilia A is scarce. Especially, data on previously untreated pediatric patients (PUPs) and minimally treated patients (MTPs) are missing.
methodsThirteen pediatric patients with haemophilia A and treatment with Emicizumab were retrospectively evaluated for Annual Bleeding Rates (ABR) pre-and post-Emicizumab treatment. Safety data and data on management of minor surgery as well as laboratory results were collected. Additionally, we describe the clinical features of two PUPs and one MTP that are included in our cohort.
resultsMedian age at initiation of Emicizumab was 5.3 (range: 0.26-17.5) years, three patients were younger than one year at initiation of treatment with Emicizumab. Median follow-up time on Emicizumab was 23.8 (range: 0.7-40) months. Total ABR (p = 0.009) as well as spontaneous (p = 0.018), traumatic (p = 0.018), and joint (p = 0.027) ABR reduced significantly post-Emicizumab transition. Safety profile was favourable as only one local site reaction occurred; no cessation of treatment was necessary. Surgery was successfully performed in three patients receiving rFVlla pre- and post-surgery. Emicizumab trough levels showed a median of 43.2 μg/ml (range: 23.9-56.8) after three doses of 3 mg/kg and 51.9 μg/ml (range: 30.4-75) at first follow-up with 1.5 mg/kg.
conclusionEmicizumab is safe and efficient in pediatric patients with and without inhibitors. More data on larger multicenter cohorts and especially on PUPs/MTPs are still needed.
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