ReviewNature reviews. Disease primers2021
Haemophilia.
Review in Nature reviews. Disease primers, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06320626 (Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study), which is not on this map. Cited by 169 papers, 3 of them syntheses that pooled 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.
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.
Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study
Who cites it
169 citing papers in PubMed, 3 syntheses or guidelines pooled it, 291 citations in OpenAlex.
- Prosthesis survival situation and complications following total hip arthroplasty in hemophilic patients: a systematic review.BMC musculoskeletal disorders · 2025Pooled it
- Liver-related aspects of valoctocogene roxaparvovec gene therapy for hemophilia A: expert guidance for clinical practice.Blood advances · 2024Guideline
- Risk of low bone mineral density in patients with haemophilia: a systematic review and meta-analysis.Journal of orthopaedic surgery and research · 2024Pooled it
- Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.Blood advances · 2025Trial
- Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.Blood advances · 2025Trial
- Matching-Adjusted Indirect Comparison of Recombinant Factor IX Albumin Fusion Protein Versus Recombinant Factor IX Fc Fusion Protein for Weekly Prophylactic Treatment of Hemophilia B.Advances in therapy · 2024Trial
- Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated XTEND-Kids Phase III Trial Patients with Haemophilia A.The patient · 2026Article
- When and How to Start Prophylaxis in Children with Hemophilia.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study.British journal of haematology · 2026Observational
- Physiologically Based Pharmacokinetic (PBPK) Modeling of FIX in Pediatric Hemophilia B: Extravascular Distribution and Dosing Optimization.Pharmaceutics · 2026Article
- A Novel GeneticTransfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Article
- FVIII-containing platelets modulate immune responses and attenuate inhibitor development in hemophilia A mice.Haematologica · 2026Article
- Reactive Oxygen Species in Cardiovascular and Hematological Diseases: Mechanisms and Therapeutic Strategies.Cureus · 2026Review
- The Impact of NEAT1 on Inflammatory Responses and Processes in Arthropathic Diseases: Latest Findings and Perspectives.Biochemical genetics · 2026Review
- Enhanced Intracellular Stability and Translation Efficiency of mRNA Drugs by a 2-arm mRNA Platform.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Longitudinal Assessment of Joint Health in Paediatric Haemophilia Using the HEAD-US Ultrasound Scoring System: The ULTRA Study.Life (Basel, Switzerland) · 2026Article
- Myeloid Cell Function and Cytokine Profiles in Paediatric Haemophilia A: Insights From FVIII and Emicizumab Prophylaxis.Journal of cellular and molecular medicine · 2026Article
- A revised classification of FVIII concentrates: rationale and novel metrics.Blood advances · 2026Article
- rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice.Gene therapy · 2026Article
- Mechanism and kinetics study of MG1113, an anti-tissue factor pathway inhibitor antibody promoting hemostasis in hemophilia.Research and practice in thrombosis and haemostasis · 2026Article
109 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 7 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease depends on the reduction of levels of FVIII or FIX, which are determined by the type of the causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark clinical characteristic, especially in untreated severe forms, is bleeding (spontaneous or after trauma) into major joints such as ankles, knees and elbows, which can result in the development of arthropathy. Intracranial bleeds and bleeds into internal organs may be life-threatening. The median life expectancy was ~30 years until the 1960s, but improved understanding of the disorder and development of efficacious therapy based on prophylactic replacement of the missing factor has caused a paradigm shift, and today individuals with haemophilia can look forward to a virtually normal life expectancy and quality of life. Nevertheless, the potential development of inhibitory antibodies to infused factor is still a major hurdle to overcome in a substantial proportion of patients. Finally, gene therapy for both types of haemophilia has progressed remarkably and could soon become a reality.
Indexed as
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What OpenQuestion holds
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.