Paolo Gresele1, Sara Orsini1, Patrizia Noris2, Emanuela Falcinelli1, Marie Christine Alessi3, Loredana Bury1, Munira Borhany4, Cristina Santoro5, Ana C Glembotsky6,7, Ana Rosa Cid8, Alberto Tosetto9, Erica De Candia10,11, Pierre Fontana12, Giuseppe Guglielmini1, Alessandro Pecci2. 1. Department of Medicine, Section of Internal and Cardiovascular Medicine, University of Perugia, Perugia, Italy. 2. Department of Internal Medicine, IRCCS Policlinico S. Matteo Foundation, University of Pavia, Pavia, Italy. 3. Centre for CardioVascular and Nutrition research (C2VN), Marseille, France. 4. Department of Hematology, Haemostasis & Thrombosis at National Institute of Blood Disease & Bone Marrow Transplantation, Karachi, Pakistan. 5. Hematology, Azienda Ospedaliera Universitaria Policlinico Umberto I, Rome, Italy. 6. Instituto de Investigaciones Médicas A. Lanari, Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires, Argentina. 7. Departamento Hematología Investigación, Consejo Nacional de Investigaciones Científicas y Tecnológicas (CONICET), Universidad de Buenos Aires, Instituto de Investigaciones Médicas (IDIM), Buenos Aires, Argentina. 8. Unidad de Hemostasia y Trombosis, Hospital Universitario y Politecnico La Fe, Valencia, Spain. 9. Hematology Department, S. Bortolo Hospital, Vicenza, Italy. 10. Hemostasis and Thrombosis Unit, Fondazione Policlinico Agostino Gemelli IRCCS, Roma, Italy. 11. Institute of Internal Medicine and Geriatrics, Università Cattolica del Sacro Cuore, Roma, Italy. 12. Platelet Group and Division of Angiology and Hemostasis, University Hospitals of Geneva, Geneva, Switzerland.
Abstract
BACKGROUND: Careful assessment of bleeding history is the first step in the evaluation of patients with mild/moderate bleeding disorders, and the use of a bleeding assessment tool (BAT) is strongly encouraged. Although a few studies have assessed the utility of the ISTH-BAT in patients with inherited platelet function disorders (IPFD) none of them was sufficiently large to draw conclusions and/or included appropriate control groups. OBJECTIVES: The aim of the present study was to test the utility of the ISTH-BAT in a large cohort of patients with a well-defined diagnosis of inherited platelets disorder in comparison with two parallel cohorts, one of patients with type-1 von Willebrand disease (VWD-1) and one of healthy controls (HC). PATIENTS/ METHODS: We enrolled 1098 subjects, 482 of whom had inherited platelet disorders (196 IPFD and 286 inherited platelet number disorders [IT]) from 17 countries. RESULTS: IPFD patients had significantly higher bleeding score (BS; median 9) than VWD-1 patients (median 5), a higher number of hemorrhagic symptoms (4 versus 3), and higher percentage of patients with clinically relevant symptoms (score > 2). The ISTH-BAT showed excellent discrimination power between IPFD and HC (0.9 < area under the curve [AUC] < 1), moderate (0.7 < AUC < 0.9) between IPFD and VWD-1 and between IPFD and inherited thrombocytopenia (IT), while it was inaccurate (AUC ≤ 0.7) in discriminating IT from HC. CONCLUSIONS: The ISTH-BAT allows to efficiently discriminate IPFD from HC, while it has lower accuracy in distinguishing IPFD from VWD-1. Therefore, the ISTH-BAT appears useful for identifying subjects requiring laboratory evaluation for a suspected IPFD once VWD is preliminarily excluded.
BACKGROUND: Careful assessment of bleeding history is the first step in the evaluation of patients with mild/moderate bleeding disorders, and the use of a bleeding assessment tool (BAT) is strongly encouraged. Although a few studies have assessed the utility of the ISTH-BAT in patients with inherited platelet function disorders (IPFD) none of them was sufficiently large to draw conclusions and/or included appropriate control groups. OBJECTIVES: The aim of the present study was to test the utility of the ISTH-BAT in a large cohort of patients with a well-defined diagnosis of inherited platelets disorder in comparison with two parallel cohorts, one of patients with type-1 von Willebrand disease (VWD-1) and one of healthy controls (HC). PATIENTS/ METHODS: We enrolled 1098 subjects, 482 of whom had inherited platelet disorders (196 IPFD and 286 inherited platelet number disorders [IT]) from 17 countries. RESULTS:IPFDpatients had significantly higher bleeding score (BS; median 9) than VWD-1patients (median 5), a higher number of hemorrhagic symptoms (4 versus 3), and higher percentage of patients with clinically relevant symptoms (score > 2). The ISTH-BAT showed excellent discrimination power between IPFD and HC (0.9 < area under the curve [AUC] < 1), moderate (0.7 < AUC < 0.9) between IPFD and VWD-1 and between IPFD and inherited thrombocytopenia (IT), while it was inaccurate (AUC ≤ 0.7) in discriminating IT from HC. CONCLUSIONS: The ISTH-BAT allows to efficiently discriminate IPFD from HC, while it has lower accuracy in distinguishing IPFD from VWD-1. Therefore, the ISTH-BAT appears useful for identifying subjects requiring laboratory evaluation for a suspected IPFD once VWD is preliminarily excluded.
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Authors: Verónica Palma-Barqueros; Nuria Revilla; Ana Sánchez; Ana Zamora Cánovas; Agustín Rodriguez-Alén; Ana Marín-Quílez; José Ramón González-Porras; Vicente Vicente; María Luisa Lozano; José María Bastida; José Rivera Journal: Int J Mol Sci Date: 2021-04-26 Impact factor: 5.923
Authors: F Atiq; J L Saes; M C Punt; K P M van Galen; R E G Schutgens; K Meijer; M H Cnossen; B A P Laros-Van Gorkom; M Peters; L Nieuwenhuizen; M J H A Kruip; J de Meris; J G van der Bom; F J M van der Meer; K Fijnvandraat; I C Kruis; W L van Heerde; H C J Eikenboom; Frank W G Leebeek; S E M Schols Journal: EClinicalMedicine Date: 2021-01-29