@article{89d006ae7eb74b31bb31deb17aff5fd6,
title = "Validation of the ISTH/SSC bleeding assessment tool for inherited platelet disorders: A communication from the Platelet Physiology SSC: Journal of Thrombosis and Haemostasis",
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.",
author = "P. Gresele and S. Orsini and P. Noris and E. Falcinelli and M.C. Alessi and L. Bury and M. Borhany and C. Santoro and A.C. Glembotsky and A.R. Cid and A. Tosetto and {De Candia}, E. and P. Fontana and G. Guglielmini and A. Pecci and P.G. Heller and G. Rodorigo and B. Lammle and A. Trinchero and R. Paolo and S. Ferrari and D. Rancitelli and A. Stolinski and A. Arulselvan and G. Lassandro and A.S. Luceros and M. Jandrot-Perrus and S. Kunishima and {Rivera Pozo}, J. and M. Lordkipanidz{\'e} and F. Melazzini and C. Falaise and A. Casonato and G. Podda and M. Kannan and K. Jurk and T. Sevivas and G. Castaman and E. Grandone and M. Fiore and P. Zuniga and Y. Henskens and K. Miyazaki and A. Dupuis and C. Hayward and C. Zaninetti and M. Abid and G. Ferrara and M.G. Mazzucconi and G. Tagariello and P. James and F. Fabris and A. Russo and N. Bermejo and M. Napolitano and J. Curnow and G. Vasiliki and B. Zieger and M. Fedor and M. Chitlur and M. Lambert and L. Barcella and B. Cosmi and P. Giordano and C. Porri and I. Eker and M.-C. Morel-Kopp and H. Deckmyn and {Frelinger A.L.}, III and P. Harrison and D. Mezzano and A.D. Mumford and investigators, {BAT-VAL study}",
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?dicas A. Lanari, Facultad de Medicina, Universidad de Buenos Aires, Buenos Aires, Argentina; Departamento Hematolog?a Investigaci?n, Consejo Nacional de Investigaciones Cient?ficas y Tecnol?gicas -CONICET-, Universidad de Buenos Aires, Instituto de Investigaciones M?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? Paris Sorbonne Cit?, UMRS1148, Paris, France), Shinji Kunishima (National Hospital Organization Nagoya Medical Center, Nagoya, Japan), Jos? Rivera Pozo (University of Murcia, Spain), Marie Lordkipanidz? (H?pital du Sacr?-C?ur de Montr?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. 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. Publisher Copyright: {\textcopyright} 2019 International Society on Thrombosis and Haemostasis",
year = "2020",
month = mar,
day = "1",
doi = "10.1111/jth.14683",
language = "English",
volume = "18",
pages = "732--739",
journal = "J. Thromb. Haemost.",
issn = "1538-7933",
publisher = "Blackwell Publishing Ltd",
number = "3",
}