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

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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.

authors

  • Gresele, Paolo
  • Orsini, Sara
  • Noris, Patrizia
  • Falcinelli, Emanuela
  • Alessi, Marie Christine
  • Bury, Loredana
  • Borhany, Munira
  • Santoro, Cristina
  • Glembotsky, Ana C
  • Cid, Ana Rosa
  • Tosetto, Alberto
  • De Candia, Erica
  • Fontana, Pierre
  • Guglielmini, Giuseppe
  • Pecci, Alessandro
  • Heller, Paula G
  • Rodorigo, Giuseppina
  • Lammle, Bernhard
  • Trinchero, Alice
  • Paolo, Radossi
  • Ferrari, Silvia
  • Rancitelli, Davide
  • Stolinski, Amy
  • Arulselvan, Abinaya
  • Lassandro, Giuseppe
  • Luceros, Analia Sanchez
  • Jandrot‐Perrus, Martine
  • Kunishima, Shinji
  • Rivera Pozo, José
  • Lordkipanidzé, Marie
  • Melazzini, Federica
  • Falaise, Céline
  • Casonato, Alessandra
  • Podda, Gianmarco
  • Kannan, Meganathan
  • Jurk, Kerstin
  • Sevivas, Teresa
  • Castaman, Giancarlo
  • Grandone, Elvira
  • Fiore, Mathieu
  • Zuniga, Pamela
  • Henskens, Yvonne
  • Miyazaki, Koji
  • Dupuis, Arnaud
  • Hayward, Catherine Pauline Ma
  • Zaninetti, Carlo
  • Abid, Madiha
  • Ferrara, Grazia
  • Mazzucconi, Maria Gabriella
  • Tagariello, Giuseppe
  • James, Paula
  • Fabris, Fabrizio
  • Russo, Alexandra
  • Bermejo, Nuria
  • Napolitano, Mariasanta
  • Curnow, Jennifer
  • Vasiliki, Gkalea
  • Zieger, Barbara
  • Fedor, Marian
  • Chitlur, Meera
  • Lambert, Michele
  • Barcella, Luca
  • Cosmi, Benilde
  • Giordano, Paola
  • Porri, Claudia
  • Eker, Ibrahim
  • Morel‐Kopp, Marie‐Christine
  • Deckmyn, Hans
  • Frelinger, Andrew L
  • Harrison, Paul
  • Mezzano, Diego
  • Mumford, Andrew D

publication date

  • March 2020