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    Poster Networking

    Hemophilia and Rare Bleeding Disorders

    Poster Networking Session

    PB1239 - Prophylaxis with Extended Half-life Products in Patients with Hemophilia A: Real-life Data of the Milan Cohort

    Tuesday, June 27, 2023
    18:30 – 19:30 ET
    Room: Exhibition

      Presenting Author(s)

    • 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

      • Co-Author(s)

    • Addolorata Truma, MD

      Resident doctor in Internal Medicine
      Università degli Studi di Milano, Department of Pathophysiology and Transplantation
      Milano, Lombardia, Italy

    • FB

      Francesco Ballardini

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

    • Maria Abbattista, PhD

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

    • Sara Arcudi, MD

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

    • Roberta Gualtierotti, MD, PhD

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

    • CM

      Claudia Mistretta

      MD
      Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi
      Milan, 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

    • Cristina Novembrino, PhD

      BSc
      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

    • 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: Prophylaxis with factor VIII (FVIII) products has become the evidence-based standard of care in patients with hemophilia A (HA). FVIII products with extended half-life (EHL) showed to be more effective compared with standard half-life products, with increased through levels and reduced number of week infusions. However, real-word data on prophylaxis with EHL products are limited.

    Aims: To evaluate prophylaxis with EHL products in patients with HA and to compare measures of efficacy and tolerability observed during prophylaxis with EHL products with those during previous prophylaxis regimens.

    Methods: We performed an observational study on patients with HA in prophylaxis with EHL FVIII with a minimum follow-up of 12 months. Exclusion criteria were previous on-demand regimen, enrollment in phase II-III trials, and dose adjustment due to weight gain or concomitant anticoagulation. Intervals of infusion, annual unit consumption for prophylaxis, annualized bleeding rates (ABRs), percentage of bleed-free patients, and trough levels were compared to the same variables during previous prophylaxis with standard half-life FVIII products. Furthermore, data were evaluated according to type of EHL product, recombinant factor VIII-Fc fusion or pegylated FVIII products.

    Results: 70 patients have been evaluated, of whom 48 met the inclusion criteria. Our switched therapeutic approach with EHL FVIII products resulted in prolonged intervals between infusions (from 2.5 to 4.0 days), increased trough levels (from 2.0 to 5.2%), and a subsequent improvement of bleeding manifestations, with the reduction of ABR (from 1.2 to 0.4) and a higher prevalence of bleed-free patients (from 58 to 79%). In addition, we observed a 19% reduction of the annual unit consumption, as shown in table. Similar results were observed stratifying patients for the type of EHL product.

    Conclusion(s): Prophylaxis with EHL products may lead to improved quality of life and treatment burden reduction.