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

Paolo Gresele; Sara Orsini; Patrizia Noris; Emanuela Falcinelli; Marie Christine Alessi; Loredana Bury; Munira Borhany; Cristina Santoro; Ana C. Glembotsky; Ana Rosa Cid; Alberto Tosetto; Erica De Candia; Pierre Fontana; Giuseppe Guglielmini; Alessandro Pecci; Federica Melazzini; Celine Falaise; Alessandra Casonato; Gianmarco Podda; Meganathan Kannan; Kerstin Jurk; Teresa Sevivas; Giancarlo Castaman; Elvira Grandone; Mathieu Fiore; Pamela Zuniga; Yvonne Henskens; Koji Miyazaki; Arnaud Dupuis; Catherine Hayward; Carlo Zaninetti; Madiha Abid; Grazia Ferrara; Maria Gabriella Mazzucconi; Giuseppe Tagariello; Paula James; Fabrizio Fabris; Alexandra Russo; Nuria Bermejo; Mariasanta Napolitano; Jennifer Curnow; Gkalea Vasiliki; Barbara Zieger; Marian Fedor; Meera Chitlur; Michele Lambert; Luca Barcella; Benilde Cosmi; Paola Giordano; Claudia Porri; BAT‐VAL study investigators

doi:10.1111/jth.14683

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

Paolo Gresele^*, Sara Orsini, Patrizia Noris, Emanuela Falcinelli, Marie Christine Alessi, Loredana Bury, Munira Borhany, Cristina Santoro, Ana C. Glembotsky, Ana Rosa Cid, Alberto Tosetto, Erica De Candia, Pierre Fontana, Giuseppe Guglielmini, Alessandro Pecci, Federica Melazzini, Celine Falaise, Alessandra Casonato, Gianmarco Podda, Meganathan KannanKerstin Jurk, Teresa Sevivas, Giancarlo Castaman, Elvira Grandone, Mathieu Fiore, Pamela Zuniga, Yvonne Henskens, Koji Miyazaki, Arnaud Dupuis, Catherine Hayward, Carlo Zaninetti, Madiha Abid, Grazia Ferrara, Maria Gabriella Mazzucconi, Giuseppe Tagariello, Paula James, Fabrizio Fabris, Alexandra Russo, Nuria Bermejo, Mariasanta Napolitano, Jennifer Curnow, Gkalea Vasiliki, Barbara Zieger, Marian Fedor, Meera Chitlur, Michele Lambert, Luca Barcella, Benilde Cosmi, Paola Giordano, Claudia Porri, BAT‐VAL study investigators

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

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

Original language	English
Pages (from-to)	732-739
Number of pages	8
Journal	Journal of Thrombosis and Haemostasis
Volume	18
Issue number	3
Early online date	16 Dec 2019
DOIs	https://doi.org/10.1111/jth.14683
Publication status	Published - Mar 2020

Keywords

bleeding assessment tool
inherited platelet disorders
bleeding diathesis
platelets
bleeding disorders
VON-WILLEBRAND-DISEASE
DIAGNOSIS
QUESTIONNAIRE
UTILITY
SYMPTOMS
SCORE
MILD
RISK

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Gresele, P., Orsini, S., Noris, P., Falcinelli, E., Alessi, M. C., Bury, L., Borhany, M., Santoro, C., Glembotsky, A. C., Rosa Cid, A., Tosetto, A., De Candia, E., Fontana, P., Guglielmini, G., Pecci, A., Melazzini, F., Falaise, C., Casonato, A., Podda, G., ... BAT‐VAL study investigators (2020). Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders: A communication from the Platelet Physiology SSC. Journal of Thrombosis and Haemostasis, 18(3), 732-739. https://doi.org/10.1111/jth.14683

@article{3fcece8a458a4813845b9d27eba6e106,

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 ",

keywords = "bleeding assessment tool, inherited platelet disorders, bleeding diathesis, platelets, bleeding disorders, VON-WILLEBRAND-DISEASE, DIAGNOSIS, QUESTIONNAIRE, UTILITY, SYMPTOMS, SCORE, MILD, RISK",

author = "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 {Rosa Cid}, Ana and Alberto Tosetto and {De Candia}, Erica and Pierre Fontana and Giuseppe Guglielmini and Alessandro Pecci and Federica Melazzini and Celine 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 Giuseppe Tagariello and Paula James and Fabrizio Fabris and Alexandra Russo and Nuria Bermejo and Mariasanta Napolitano and Jennifer Curnow and Gkalea Vasiliki and Barbara Zieger and Marian Fedor and Meera Chitlur and Michele Lambert and Luca Barcella and Benilde Cosmi and Paola Giordano and Claudia Porri and {BAT‐VAL study investigators}",

note = "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 M{\'e}dicas A. Lanari, Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires, Argentina; Departamento Hematolog{\'i}a Investigaci{\'o}n, Consejo Nacional de Investigaciones Cient{\'i}ficas y Tecnol{\'o}gicas -CONICET-, Universidad de Buenos Aires, Instituto de Investigaciones M{\'e}dicas -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, Universit{\'e} Paris Sorbonne Cit{\'e}, UMRS1148, Paris, France), Shinji Kunishima (National Hospital Organization Nagoya Medical Center, Nagoya, Japan), Jos{\'e} Rivera Pozo (University of Murcia, Spain), Marie Lordkipanidz{\'e} (H{\^o}pital du Sacr{\'e}-C{\oe}ur de Montr{\'e}al, 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: {\textcopyright} 2019 International Society on Thrombosis and Haemostasis",

year = "2020",

month = mar,

doi = "10.1111/jth.14683",

language = "English",

volume = "18",

pages = "732--739",

journal = "Journal of Thrombosis and Haemostasis",

issn = "1538-7933",

publisher = "Wiley",

number = "3",

}

Gresele, P, Orsini, S, Noris, P, Falcinelli, E, Alessi, MC, Bury, L, Borhany, M, Santoro, C, Glembotsky, AC, Rosa Cid, A, Tosetto, A, De Candia, E, Fontana, P, Guglielmini, G, Pecci, A, Melazzini, F, Falaise, C, Casonato, A, Podda, G, Kannan, M, Jurk, K, Sevivas, T, Castaman, G, Grandone, E, Fiore, M, Zuniga, P, Henskens, Y, Miyazaki, K, Dupuis, A, Hayward, C, Zaninetti, C, Abid, M, Ferrara, G, Mazzucconi, MG, Tagariello, G, James, P, Fabris, F, Russo, A, Bermejo, N, Napolitano, M, Curnow, J, Vasiliki, G, Zieger, B, Fedor, M, Chitlur, M, Lambert, M, Barcella, L, Cosmi, B, Giordano, P, Porri, C & BAT‐VAL study investigators 2020, 'Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders: A communication from the Platelet Physiology SSC', Journal of Thrombosis and Haemostasis, vol. 18, no. 3, pp. 732-739. https://doi.org/10.1111/jth.14683

TY - JOUR

T1 - Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders

T2 - A communication from the Platelet Physiology SSC

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 - Rosa Cid, Ana

AU - Tosetto, Alberto

AU - De Candia, Erica

AU - Fontana, Pierre

AU - Guglielmini, Giuseppe

AU - Pecci, Alessandro

AU - Melazzini, Federica

AU - Falaise, Celine

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 - Tagariello, Giuseppe

AU - James, Paula

AU - Fabris, Fabrizio

AU - Russo, Alexandra

AU - Bermejo, Nuria

AU - Napolitano, Mariasanta

AU - Curnow, Jennifer

AU - Vasiliki, Gkalea

AU - Zieger, Barbara

AU - Fedor, Marian

AU - Chitlur, Meera

AU - Lambert, Michele

AU - Barcella, Luca

AU - Cosmi, Benilde

AU - Giordano, Paola

AU - Porri, Claudia

AU - BAT‐VAL study investigators

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

Y1 - 2020/3

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

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

KW - bleeding assessment tool

KW - inherited platelet disorders

KW - bleeding diathesis

KW - platelets

KW - bleeding disorders

KW - VON-WILLEBRAND-DISEASE

KW - DIAGNOSIS

KW - QUESTIONNAIRE

KW - UTILITY

KW - SYMPTOMS

KW - SCORE

KW - MILD

KW - RISK

U2 - 10.1111/jth.14683

DO - 10.1111/jth.14683

M3 - Article

C2 - 31750621

SN - 1538-7933

VL - 18

SP - 732

EP - 739

JO - Journal of Thrombosis and Haemostasis

JF - Journal of Thrombosis and Haemostasis

IS - 3

ER -

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

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