European Management of Glanzmann's Thrombasthenia: A Survey of Current Clinical Practice

Authors: Fiore, M; Artoni, A; Klamroth, R; Mathias, M; Schutgens, R; D’Oiron, R

Affiliations: Department of Haematology, University Hospital of Bordeaux, Pessac, France. Biology of Cardiovascular Disease, Inserm U1034, Pessac, France. French Reference Centre For Inherited Platelet Disorders, University Hospital of Bordeaux, Pessac, France. Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy. Department of Internal Medicine, Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, Germany. Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. Department of Benign Hematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht and University Utrecht, Utrecht, Netherlands. Centre De Référence de L’hémophilie Et Des Maladies Hémorragiques Rares, Hôpital Bicêtre AP-HP ; and INSERM Hémostase Inflammation Thrombose HITH U1176, Université Paris-Saclay, Le Kremlin-Bicêtre, France.

Publication: Hemophilia; 2025

ABSTRACT: INTRODUCTION: Glanzmann’s thrombasthenia is a rare inherited platelet disorder characterized by a lack of platelet aggregation. Patients tend to be diagnosed in early childhood with treatment strategies involving a multifaceted approach to prevent and manage bleeding episodes. Unfortunately, there is currently no European consensus regarding the management of GT. AIM: This initiative aimed to gain an understanding of current clinical management of GT across Europe, with the aim of aligning best practice and improving patient outcomes. METHODS: The authors, on behalf of the EAHAD Glanzmann Working Group, administered an online survey of 57 questions to European hematologists currently involved in the management of patients with GT. The survey covered topics related to diagnosis, treatment access and selection, immunization, peri-operative management and use of second-line therapies. RESULTS: Responses reflected physician consensus around some topics, including peri-operative treatment, use of recombinant factor VIIa, and concerns around antibody development. However, more varied responses were received on topics such as antibody screening (anti-αIIbβ3 antibodies screening conducted by ≤53% of respondents in all countries of interest except France), access to HLA-matched platelet concentrates (none or limited for 55% of respondents) and duration of platelet transfusions for major surgery (13%-31% for 1, 2, 3 and 4 or more days of transfusions). CONCLUSION: Establishing comprehensive guidelines to manage GT will enhance patient outcomes by ensuring patients receive high-quality and effective care as well as standardize care across different healthcare settings.