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    Poster Networking

    Hemophilia and Rare Bleeding Disorders

    Poster Networking Session

    PB0683 - Patients at higher risk of spontaneous joint bleeding during emicizumab prophylaxis: a study on global coagulation assays and arthropathy scores

    Monday, June 26, 2023
    18:30 – 19:30 ET
    Room: Exhibition

      Presenting Author(s)

    • Sara Arcudi, MD

      Academic researcher, MD, PhD
      Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi
      Milan, Lombardia, Italy

      • Co-Author(s)

    • Roberta Gualtierotti, MD, PhD

      Associate Professor
      Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi
      Milan, Lombardia, Italy

    • ES

      Erica Scalambrino, MA

      Collaboratore Professionale di Ricerca
      Fondazione IRCCS Cà Grand Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
      Milano, Lombardia, Italy

    • MC

      Marigrazia Clerici

      BSc
      Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
      Milano, Lombardia, Italy

    • SH

      Shermarke Hassan

      Academic Researcher
      Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands
      Milano, Lombardia, Italy

    • GN

      Gabriella Nicolò

      Research Nurse
      Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, Department of Healthcare Professions, Milan, Italy
      Milano, Lombardia, Italy

    • Alessandro Ciavarella, MD (he/him/his)

      Specialized Doctor
      Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi
      Milan, Lombardia, Italy

    • VB

      Valentina Begnozzi

      Physioterapist
      Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
      Milano, Lombardia, Italy

    • EB

      Elena Boccalandro

      Physiotherapist
      Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
      Milan, Lombardia, Italy

    • SS

      Simona Maria Siboni

      Hematologist
      Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
      Milan, Lombardia, Italy

    • Cristina Novembrino, PhD

      BSc
      Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
      Milan, Lombardia, Italy

    • CV

      Carla Valsecchi

      Biologist
      Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
      Milano, Lombardia, Italy

    • Flora Peyvandi, MD, PhD (she/her/hers)

      Director
      IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy
      Milano, Lombardia, Italy

    Background: Emicizumab was the first non-replacement product approved for patients with severe hemophilia A (HA). Notwithstanding its great efficacy in reducing bleeding, some patients still experience spontaneous joint bleeding (SJB), but no method is yet available to identify patients at higher spontaneous bleeding risk.

    Aims: To evaluate whether non-activated thromboelastography (NATEM), thrombin generation assay (TGA) and arthropathy scores during emicizumab prophylaxis could distinguish patients at higher risk of SJB development.

    Methods: Consecutive HA patients on emicizumab prophylaxis were enrolled. Blood samples were drawn at baseline and at weeks 5, 10, 20 and 50 of emicizumab prophylaxis in new starters and at steady state in patients already on maintenance. TGA was assessed upon addition of tissue-factor to plasma and synthetic phospholipids. NATEM was performed in citrated blood after addition of CaCl2. Hemophilic arthropathy was assessed with the hemophilia joint health (HJHS) and the hemophilia early arthropathy detection ultrasound (HEAD-US) scores. Cox proportional hazards model was used to evaluate the association between clinical and laboratory parameters and the bleeding outcome. A model capable of predicting the spontaneous bleeding risk was investigated with ROC analysis.

    Results: Forty patients with HA on emicizumab were enrolled, 10 patients developed spontaneous joint bleeding. The median total ABR was 0.70 (95%CI 0.51-0.89), median spontaneous joint ABR was 0.30 (95%CI 0.18-0.43). TGA and NATEM parameters were not associated with the bleeding outcome (table 1). ROC analysis revealed that the synovitis and the HEAD-US scores together resulted to be good predictors of SJB development (AUC 0.84, figure 1), whereas a lower emicizumab plasma concentration (AUC 0.62) and a greater degree of physical exertion (AUC 0.65) were not associated with the bleeding outcome.

    Conclusion(s): The degree of hemophilic arthropathy was associated with the bleeding outcome, while global coagulation assays failed in distinguishing patients at higher risk of SJB development.