Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders: A communication from the Platelet Physiology SSC. (16th December 2019)

• Record Type: Journal Article
• Authors: Gresele, Paolo; Orsini, Sara; Noris, Patrizia; Falcinelli, Emanuela; Alessi, Marie Christine; Bury, Loredana; Borhany, Munira; Santoro, Cristina; Glembotsky, Ana C.; Cid, Ana Rosa; Tosetto, Alberto; De Candia, Erica; Fontana, Pierre; Guglielmini, Giuseppe; Pecci, Alessandro
• Other Names: Heller Paula G. investigator.; Rodorigo Giuseppina investigator.; Lammle Bernhard investigator.; Trinchero Alice investigator.; Paolo Radossi investigator.; Ferrari Silvia investigator.; Rancitelli Davide investigator.; Stolinski Amy investigator.; Arulselvan Abinaya investigator.; Lassandro Giuseppe investigator.; Luceros Analia Sanchez investigator.; Jandrot‐Perrus Martine investigator.; Kunishima Shinji investigator.; Rivera Pozo José investigator.; Lordkipanidzé Marie investigator.; Melazzini Federica investigator.; Falaise Céline investigator.; Casonato Alessandra investigator.; Podda Gianmarco investigator.; Kannan Meganathan investigator.; Jurk Kerstin investigator.; Sevivas Teresa investigator.; Castaman Giancarlo investigator.; Grandone Elvira investigator.; Fiore Mathieu investigator.; Zuniga Pamela investigator.; Henskens Yvonne investigator.; Miyazaki Koji investigator.; Dupuis Arnaud investigator.; Hayward Catherine investigator.; Zaninetti Carlo investigator.; Abid Madiha investigator.; Ferrara Grazia investigator.; Mazzucconi Maria Gabriella investigator.; Tagariello Giuseppe investigator.; James Paula investigator.; Fabris Fabrizio investigator.; Russo Alexandra investigator.; Bermejo Nuria investigator.; Napolitano Mariasanta investigator.; Curnow Jennifer investigator.; Vasiliki Gkalea investigator.; Zieger Barbara investigator.; Fedor Marian investigator.; Chitlur Meera investigator.; Lambert Michele investigator.; Barcella Luca investigator.; Cosmi Benilde investigator.; Giordano Paola investigator.; Porri Claudia investigator.; Eker Ibrahim investigator.; Morel‐Kopp Marie‐Christine investigator.; Deckmyn Hans investigator.; Frelinger Andrew L. investigator.; Harrison Paul investigator.; Mezzano Diego investigator.; Mumford Andrew D. investigator.;
• Is Part Of: Journal of thrombosis and haemostasis. Volume 18:Number 3(2020)
• Page Start: 732
• Page End: 739
• Publication Date: 2019-12-16
• Subjects: bleeding assessment tool -- inherited platelet disorders -- bleeding diathesis -- platelets -- bleeding disorders; Thrombosis -- Periodicals; Hemostasis -- Periodicals; Blood coagulation disorders -- Periodicals; 616.1
• DOI: 10.1111/jth.14683
• Languages: English
• ISSNs: 1538-7933

Abstract

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.