ArticleBlood advances2020
Bleeding severity in patients with rare bleeding disorders: real-life data from the RBiN study.
Article in Blood advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03347591 (Rare Bleeding Disorders in the Netherlands), which is not on this map. Cited by 23 papers.
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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.
Rare Bleeding Disorders in the Netherlands
Who cites it
23 citing papers in PubMed, 48 citations in OpenAlex.
- Genetic and hemostatic characterization of PAI-1 deficiency and isolated hyperfibrinolysis: data from the RBiN study.Research and practice in thrombosis and haemostasis · 2026Article
- Optimizing Surgical Approaches for Patients with Inherited Factor VII Deficiency.Thrombosis and haemostasis · 2026Article
- Focus on Women with Hemophilia and Carriers: How to Address All the Existing Gaps in Diagnosis and Care.Journal of clinical medicine · 2026Review
- A multicenter prospective observational cohort study to improve diagnostic yield and clinical management of persons with a Bleeding Disorder of Unknown Cause (BDUC-iN study protocol).Research and practice in thrombosis and haemostasis · 2026Observational
- Exploration of the Pathogenic Mechanism of the Factor XIII A Subunit in a Patient With Congenital Factor XIII Deficiency.Molecular genetics & genomic medicine · 2026Article
- Genetic mutations associated with congenital fibrinogen disorders: global distribution and clinical outcomes.Journal of thrombosis and thrombolysis · 2026Review
- Clinical and Genetic Heterogeneity of Factor XI Deficiency: Insights from a Southern Italian Cohort.International journal of molecular sciences · 2025Article
- Health-related quality of life in rare bleeding disorders: results from the Rare Bleeding Disorders in the Netherlands study.Research and practice in thrombosis and haemostasis · 2025Article
- Gynecologic and obstetric complications in women with congenital fibrinogen disorders: insights from the Prospective Rare Bleeding Disorders Database.Research and practice in thrombosis and haemostasis · 2025Article
- Article
- Enhanced thrombin and plasmin generation profiles in alpha-2-antiplasmin-deficient patients: Data from the Rare Bleeding disorders in the Netherlands study.Research and practice in thrombosis and haemostasis · 2024Article
- Targeted exome analysis in patients with rare bleeding disorders: data from the Rare Bleeding Disorders in the Netherlands study.Research and practice in thrombosis and haemostasis · 2024Article
- Psychometrics of patient-reported outcomes measurement information system in von Willebrand disease, inherited platelet function disorders, and rare bleeding disorders.Research and practice in thrombosis and haemostasis · 2024Article
- Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database.Blood advances · 2024Article
- Obstetric and perioperative management of patients with factor XI deficiency: a retrospective observational study.Blood advances · 2023Observational
- VHrare study: Prevalence, clinical features and management of severe rare bleeding disorders in a large cohort.EJHaem · 2023Review
- Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders.Expert review of hematology · 2023Article
- Screening platelet function in blood donors.Transfusion · 2022Article
- SYMPHONY consortium: Orchestrating personalized treatment for patients with bleeding disorders.Journal of thrombosis and haemostasis : JTH · 2022Article
- Treatment of patients with rare bleeding disorders in the Netherlands: Real-life data from the RBiN study.Journal of thrombosis and haemostasis : JTH · 2022Article
Corrections and comments
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Authors and funding
11 authors at 7 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Patients with hereditary rare bleeding disorders (RBDs) present with diverse hemorrhagic symptoms. Correlation between factor activity levels and clinical bleeding severity is poor for most RBDs. Threshold factor activity levels have been previously described in relation to bleeding severity but have not yet been validated. The Rare Bleeding Disorders in the Netherlands (RBiN) study is a nationwide cross-sectional study of patients registered in all 6 Dutch Haemophilia Treatment Centers with a known RBD and who are age 1 to 99 years. Bleeding scores were determined, and laboratory and clinical data were extracted from patient files. In all, 263 patients were included, of whom 202 (77%) attended the scheduled study visit. The median International Society of Thrombosis and Haemostasis (ISTH) bleeding assessment tool (BAT) score was 9. Correlations between baseline factor activity levels and ISTH BAT scores were strong for deficiencies in factor II (FII) (r = -0.792) and FX (r = -0.838) and were moderate for deficiencies of fibrinogen (r = -0.683), FV (r = -0.623), FVII (r = -0.516), FXIII (r = -0.516), and α2-antiplasmin (r = -0.594). There was no correlation for FXI deficiency (r = -0.218). The RBD BAT identified more women (94% vs 83%) and children (100% vs 71%) with an RBD than the ISTH BAT did. Importantly, 48% of patients had more severe bleeding than predicted for their baseline factor activity level. In addition, 34% of patients were predicted to be asymptomatic, but they actually had grade 2 (31%) or 3 (3%) bleeding. Bleeding severity in patients with RBDs is more pronounced than previously anticipated. The previously determined threshold factor activity levels to ensure no (spontaneous) bleeding in patients with an RBD are inaccurate. This trial was registered at www.clinicaltrials.gov as #NCT03347591.
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