TY - JOUR
T1 - Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders
T2 - A communication from the Platelet Physiology SSC
AU - BAT-VAL study investigators
AU - Gresele, Paolo
AU - Orsini, Sara
AU - Noris, Patrizia
AU - Falcinelli, Emanuela
AU - Alessi, Marie Christine
AU - Bury, Loredana
AU - Borhany, Munira
AU - Santoro, Cristina
AU - Glembotsky, Ana C.
AU - Cid, Ana Rosa
AU - Tosetto, Alberto
AU - De Candia, Erica
AU - Fontana, Pierre
AU - Guglielmini, Giuseppe
AU - Pecci, Alessandro
AU - Heller, Paula G.
AU - Rodorigo, Giuseppina
AU - Lammle, Bernhard
AU - Trinchero, Alice
AU - Paolo, Radossi
AU - Ferrari, Silvia
AU - Rancitelli, Davide
AU - Stolinski, Amy
AU - Arulselvan, Abinaya
AU - Lassandro, Giuseppe
AU - Luceros, Analia Sanchez
AU - Jandrot-Perrus, Martine
AU - Kunishima, Shinji
AU - Rivera Pozo, José
AU - Lordkipanidzé, Marie
AU - Melazzini, Federica
AU - Falaise, Céline
AU - Casonato, Alessandra
AU - Podda, Gianmarco
AU - Kannan, Meganathan
AU - Jurk, Kerstin
AU - Sevivas, Teresa
AU - Castaman, Giancarlo
AU - Grandone, Elvira
AU - Fiore, Mathieu
AU - Zuniga, Pamela
AU - Henskens, Yvonne
AU - Miyazaki, Koji
AU - Dupuis, Arnaud
AU - Hayward, Catherine
AU - Zaninetti, Carlo
AU - Abid, Madiha
AU - Ferrara, Grazia
AU - Mazzucconi, Maria Gabriella
AU - Chitlur, Meera
N1 - Publisher Copyright:
© 2019 International Society on Thrombosis and Haemostasis
PY - 2020/3/1
Y1 - 2020/3/1
N2 - 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.
AB - 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.
KW - bleeding assessment tool
KW - bleeding diathesis
KW - bleeding disorders
KW - inherited platelet disorders
KW - platelets
UR - http://www.scopus.com/inward/record.url?scp=85076790964&partnerID=8YFLogxK
U2 - 10.1111/jth.14683
DO - 10.1111/jth.14683
M3 - Article
C2 - 31750621
AN - SCOPUS:85076790964
SN - 1538-7933
VL - 18
SP - 732
EP - 739
JO - Journal of Thrombosis and Haemostasis
JF - Journal of Thrombosis and Haemostasis
IS - 3
ER -