Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders: A communication from the Platelet Physiology SSC

BAT-VAL study investigators

Research output: Contribution to journalArticle

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.

Original languageEnglish
JournalJournal of Thrombosis and Haemostasis
DOIs
Publication statusAccepted/In press - Jan 1 2019

Keywords

  • bleeding assessment tool
  • bleeding diathesis
  • bleeding disorders
  • inherited platelet disorders
  • platelets

ASJC Scopus subject areas

  • Hematology

Cite this

@article{efb619c273f7487f8f3b8f01276a5022,
title = "Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders: A communication from the Platelet Physiology SSC",
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.",
keywords = "bleeding assessment tool, bleeding diathesis, bleeding disorders, inherited platelet disorders, platelets",
author = "{BAT-VAL study investigators} and Paolo Gresele and Sara Orsini and Patrizia Noris and Emanuela Falcinelli and Alessi, {Marie Christine} and Loredana Bury and Munira Borhany and Cristina Santoro and Glembotsky, {Ana C.} and Cid, {Ana Rosa} and Alberto Tosetto and {De Candia}, Erica and Pierre Fontana and Giuseppe Guglielmini and Alessandro Pecci and Heller, {Paula G.} and Giuseppina Rodorigo and Bernhard Lammle and Alice Trinchero and Radossi Paolo and Silvia Ferrari and Davide Rancitelli and Amy Stolinski and Abinaya Arulselvan and Giuseppe Lassandro and Luceros, {Analia Sanchez} and Martine Jandrot-Perrus and Shinji Kunishima and {Rivera Pozo}, Jos{\'e} and Marie Lordkipanidz{\'e} and Federica Melazzini and C{\'e}line Falaise and Alessandra Casonato and Gianmarco Podda and Meganathan Kannan and Kerstin Jurk and Teresa Sevivas and Giancarlo Castaman and Elvira Grandone and Mathieu Fiore and Pamela Zuniga and Yvonne Henskens and Koji Miyazaki and Arnaud Dupuis and Catherine Hayward and Carlo Zaninetti and Madiha Abid and Grazia Ferrara and Mazzucconi, {Maria Gabriella} and Mariasanta Napolitano",
year = "2019",
month = "1",
day = "1",
doi = "10.1111/jth.14683",
language = "English",
journal = "Journal of Thrombosis and Haemostasis",
issn = "1538-7933",
publisher = "Wiley-Blackwell",

}

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 - Napolitano, Mariasanta

PY - 2019/1/1

Y1 - 2019/1/1

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

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UR - http://www.scopus.com/inward/citedby.url?scp=85076790964&partnerID=8YFLogxK

U2 - 10.1111/jth.14683

DO - 10.1111/jth.14683

M3 - Article

C2 - 31750621

AN - SCOPUS:85076790964

JO - Journal of Thrombosis and Haemostasis

JF - Journal of Thrombosis and Haemostasis

SN - 1538-7933

ER -