Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders |
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| Authors: | F. PEYVANDI,R. PALLA,M. MENEGATTI,S. M. SIBONI,S. HALIMEH,B. FAESER,H. PERGANTOU,H. PLATOKOUKI,P. GIANGRANDE,K. PEERLINCK,T. CELKAN,N. OZDEMIR,C. BIDLINGMAIER,J. INGERSLEV,M. GIANSILY‐BLAIZOT,J. F. SCHVED,R. GILMORE,A. GADISSEUR,M. BENEDIK‐DOLNI AR,L. KITANOVSKI,D. MIKOVIC,K. M. MUSALLAM,F. R. ROSENDAAL |
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| Affiliation: | 1. U.O.S. Dipartimentale per la Diagnosi e la Terapia delle Coagulopatie, A. Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione I.R.C.C.S. Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano and Luigi Villa Foundation, Milan, Italy;2. MVZ Labor Duisburg GmbH, Duisburg, Germany;3. Haemophillia Center, Haemostasis Unit, Agia Sofia Children’s Hospital, Athens, Greece;4. Oxford Haemophilia and Thrombosis Centre, Nuffield Department of Clinical Medicine, University of Oxford, Oxford, UK;5. Hemofilie Centrum Leuven, Katholieke Universiteit, Leuven, Belgium;6. Department of Pediatric Hematology‐Oncology, Cerrahpasa Medical Faculty of Istanbul University, Istanbul, Turkey;7. Pediatric Hemophilia and Thrombosis Centre, Dr von Hauner’s Children’s University Hospital, Munich, Germany;8. Centre for Hemophilia and Thrombosis, Department of Clinical Biochemistry, University Hospital Skejbi, Aarhus, Denmark;9. Hopital Saint Eloi, Montpellier, France;10. National Centre for Hereditary Coagulation Disorders, St James’s Hospital, Dublin, Ireland;11. Antwerp University Hospital UZA, Edegem, Belgium;12. National Haemophilia Centre, Department of Pediatrics, University Medical Centre, Ljubljana, Slovenia;13. Haemostasis Department and Haemophilia Center, Blood Transfusion Institute of Serbia, Belgrade, Serbia;14. Department of Thrombosis and Hemostasis and Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands |
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| Abstract: | Summary. Background: The European Network of Rare Bleeding Disorders (EN‐RBD) was established to bridge the gap between knowledge and practise in the care of patients with RBDs. Objectives: To explore the relationship between coagulation factor activity level and bleeding severity in patients with RBDs. Patients/Methods: Cross‐sectional study using data from 489 patients registered in the EN‐RBD. Coagulation factor activity levels were retrieved. Clinical bleeding episodes were classified into four categories according to severity. Results: The mean age of patients at data collection was 31 years (range, 7 months to 95 years), with an equal sex distribution. On linear regression analysis, there was a strong association between coagulation factor activity level and clinical bleeding severity for fibrinogen, factor (F) X, FXIII, and combined FV and FVIII deficiencies. A weaker association was present for FV and FVII deficiencies. There was no association between coagulation factor activity level and clinical bleeding severity for FXI. The coagulation factor activity levels that were necessary for patients to remain asymptomatic were: fibrinogen, > 100 mg dL?1; FV, 12 U dL?1; combined FV + VIII, 43 U dL?1; FVII, 25 U dL?1; FX, 56 U dL?1; FXI, 26 U dL?1; FXIII, 31 U dL?1. Moreover, coagulation factor activity levels that corresponded with Grade III bleeding were: undetectable levels for fibrinogen, FV and FXIII, < 15 U dL?1 for combined FV + VIII; < 8 U dL?1 for FVI; < 10 U dL?1 for FX; and < 25 U dL?1 for FXI. Conclusions: There is a heterogeneous association between coagulation factor activity level and clinical bleeding severity in different RBDs. A strong association is only observed in fibrinogen, FX and FXIII deficiencies. |
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| Keywords: | bleeding episode coagulant activity phenotype rare bleeding disorder |
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