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

ORSINI, SARA; ALESSI, MARIE CHRISTINE; SANTORO, CRISTINA; TOSETTO, ALBERTO; GUGLIELMINI, GIUSEPPE; RODORIGO, GIUSEPPINA; PAOLO, RADOSSI; STOLINSKI, AMY; LUCEROS, ANALIA SANCHEZ; RIVERA POZO, JOSÉ; FALAISE, CÉLINE; KANNAN, MEGANATHAN; CASTAMAN, GIANCARLO; ZUNIGA, PAMELA; DUPUIS, ARNAUD; ABID, MADIHA; TAGARIELLO, GIUSEPPE; RUSSO, ALEXANDRA; CURNOW, JENNIFER; FEDOR, MARIAN; BARCELLA, LUCA; PORRI, CLAUDIA; DECKMYN, HANS; MEZZANO, DIEGO; GRESELE, PAOLO; FALCINELLI, EMANUELA; BORHANY, MUNIRA; CID, ANA ROSA; FONTANA, PIERRE; HELLER, PAULA G.; TRINCHERO, ALICE; RANCITELLI, DAVIDE; LASSANDRO, GIUSEPPE; KUNISHIMA, SHINJI; MELAZZINI, FEDERICA; PODDA, GIANMARCO; SEVIVAS, TERESA; FIORE, MATHIEU; MIYAZAKI, KOJI; ZANINETTI, CARLO; MAZZUCCONI, MARIA GABRIELLA; FABRIS, FABRIZIO; NAPOLITANO, MARIASANTA; ZIEGER, BARBARA; LAMBERT, MICHELE; GIORDANO, PAOLA; MOREL?KOPP, MARIE?CHRISTINE; HARRISON, PAUL; NORIS, PATRIZIA; BURY, LOREDANA; GLEMBOTSKY, ANA C.; DE CANDIA, ERICA; PECCI, ALESSANDRO; LAMMLE, BERNHARD; FERRARI, SILVIA; ARULS

JOURNAL OF THROMBOSIS AND HAEMOSTASIS

WILEY-BLACKWELL PUBLISHING, INC

Año: 2020 vol. 18 p. 732 - 739

1538-7933

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.