Coagulation Factor Activity Level and Clinical Bleeding Severity in Rare Bleeding Disorders: Results From the European Network of Rare Bleeding Disorders (EN-RBD),

Blood - Tập 118 - Trang 3312 - 2011
Flora Peyvandi1, Roberta Palla1, Marzia Menegatti1, Simona M Siboni1, Susan Halimeh2, Britta Faeser2, Helen Pergantou3, Heleni Platokouki3, Paul Giangrande4, Kathelijne Peerlink5, Tiraje Celkan6, Nihal Ozdemir6, Christoph Bidlingmaier7, Jorgen Ingerslev8, Muriel Giansily-Blaizot9, Jean-Francois Schved9, Ruth Gilmore10, Alain P. Gadisseur11, Majda Benedik-Dolnicar12, Lidija Kitanovski12
1U.O.S. Dipartimentale per la Diagnosi e la Terapia delle Coagulopatie, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda - Ospedale Maggiore Policlinico, Università degli Studi di Milano and Luigi Villa Foundation, Milan, Italy
2Hemophilia treatment center, MVZ Duisburg, Duisburg, Germany
3Haemophilia Centre, Haemostasis Unit, “Aghia Sophia” Children's Hospital, Athens, Greece
4Oxford Haemophilia & Thrombosis Centre, Nuffield Department of Clinical Medicine, University of Oxford, Oxford, United Kingdom
5Hemofilie Centrum Leuven, Katholieke Universiteit, Leuven, Belgium
6Department of Pediatric Hematology-Oncology, Cerrahpasa Medical Faculty of Istanbul University, Istanbul, Turkey
7Pediatrics, Univ. Childrens Hospital, Munich, Germany
8Skejby University Hospital, Centre for Haemophilia and Thrombosis, Aarhus, Denmark
9Laboratoire d'hematologie, Hospital Saint Eloi, Montpellier, France
10National Centre for Hereditary Coagulation Disorders, St James's Hospital, Dublin, Ireland
11Department of Haematology, Antwerp University Hospital UZA, Edegem, Belgium
12Department of Pediatrics, National Haemophilia Centre, University Medical Centre, Ljubljana, Slovenia

Tóm tắt

Abstract Abstract 3312 The European Network of Rare Bleeding Disorders (EN-RBD) was established to bridge the gap between knowledge and practice in the care of patients with RBDs (rare bleeding disorders frequency <1/0.5-2M). The aim of this first report was to explore the relationship between the coagulation factor activity levels and clinical bleeding severity in patients with RBDs. A total of 592 records on patients with RBDs were cross-sectionally collected over a period of three years. Data on clinical bleeding episodes were available for 495 patients and were classified into four categories according to severity. The mean age of patients was 31 years (range, 7 months-95 years), with 51% being females. On linear regression analysis, there was a strong association between coagulation factor activity level and clinical bleeding severity for fibrinogen, factor (F)FX, 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. It is of clinical relevance to identify levels that are not associated with spontaneous major bleeding, which was done by constructing receiver operating characteristic curves. These levels were highest for FXIII deficiency (25 U/dl), followed by FV+VII (15 U/dl), FVII (15 U/dl), FX (5 U/dl), and FV (1 U/dl) deficiencies. For fibrinogen deficiency, a factor activity level of 20 mg/dl is needed to ensure absence of major spontaneous bleeding. There is a clear relation between coagulation factor activity level and clinical bleeding severity in RBDs, which is different for the missing clotting factor. Disclosures: Peyvandi: NovoNordisk, CSL Behring: Honoraria. Bidlingmaier:CSL Behring, Bayer Schering: Research Funding; CSL Behring, NovoNordisk, Pfizer, Bayer Schering: Membership on an entity's Board of Directors or advisory committees; Baxter, Biotest, CSL Behring, NovoNordisk, Pfizer, Bayer Schering: Honoraria. Schved:LFB, CSL Behring, Novonordisk, Bayer Schering: Research Funding.

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Blood

Tập 118

3312

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