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 - Funding Information:
LB and EF were supported by a scholarship grant from Fondazione Umberto Veronesi. This study was supported in part by a Telethon grant (GGP15063) to PG. The authors thank Dr. Francesco Rodeghiero (Department of Cell Therapy and Hematology, San Bortolo Hospital, Vicenza, Italy) for his critical review of the manuscript. The contribution of Paula G. Heller (Instituto de Investigaciones Médicas A. Lanari, Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires, Argentina; Departamento Hematología Investigación, Consejo Nacional de Investigaciones Científicas y Tecnológicas -CONICET-, Universidad de Buenos Aires, Instituto de Investigaciones Médicas -IDIM-, Buenos Aires, Argentina), Giuseppina Rodorigo (S.Orsola -Malpighi University Hospital, University of Bologna, Italy), Bernhard Lammle and Alice Trinchero (University Medical Center, Mainz, Germany), Radossi Paolo (Castelfranco Veneto Hospital, Italy), Silvia Ferrari and Davide Rancitelli (University of Padua, Italy), Amy Stolinski (Children's Hospital of Michigan, Detroit, Michigan, USA), Abinaya Arulselvan (Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA), Giuseppe Lassandro (University of Bari, Italy), and Analia Sanchez Luceros (Hospital Italiano, Rosario, Argentina) for patient enrollment is kindly acknowledged. The authors acknowledge Martine Jandrot-Perrus (Inserm, Université Paris Sorbonne Cité, UMRS1148, Paris, France), Shinji Kunishima (National Hospital Organization Nagoya Medical Center, Nagoya, Japan), José Rivera Pozo (University of Murcia, Spain), Marie Lordkipanidzé (Hôpital du Sacré-Cœur de Montréal, Montreal, Quebec, Canada) that were members of the SSC Platelet Physiology when the study was promoted.
Funding Information:
LB and EF were supported by a scholarship grant from Fondazione Umberto Veronesi. This study was supported in part by a Telethon grant (GGP15063) to PG. The authors thank Dr. Francesco Rodeghiero (Department of Cell Therapy and Hematology, San Bortolo Hospital, Vicenza, Italy) for his critical review of the manuscript.
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
VL - 18
SP - 732
EP - 739
JO - Journal of Thrombosis and Haemostasis
JF - Journal of Thrombosis and Haemostasis
SN - 1538-7933
IS - 3
ER -