{
  "abstract": "Background and Importance Emicizumab is a humanised bispecific monoclonal antibody that mimics the cofactor function of activated factor VIII and is primarily indicated for the prophylactic treatment of patients with haemophilia A, irrespective of the presence of inhibitors. While its use in other hereditary bleeding disorders, such as von Willebrand disease, remains off-label and limited, it is increasingly being explored as a therapeutic alternative. We report our real-world experience from a tertiary care centre, highlighting a notable case of type III von Willebrand disease with an inhibitor, in which the use of emicizumab yielded promising clinical outcomes.Aim and Objectives To evaluate the efficacy, safety, and clinical applicability of emicizumab in patients with hereditary coagulopathies, with a particular focus on a case of type III von Willebrand disease with an inhibitor.Material and Methods This is a single-centre, retrospective observational study involving eight patients treated with emicizumab between December 2019 and May 2025. Clinical, genetic, and follow-up variables were collected, including diagnosis, age at treatment initiation, presence of inhibitors, history of bleeding episodes, post-treatment bleeding events, surgical or invasive interventions and adverse reactions.Results The cohort included seven patients with severe haemophilia A (one with an inhibitor) and one patient with type III von Willebrand disease with an inhibitor. The mean age at treatment initiation was 22 years (range: 5–40). All patients had a history of clinically significant bleeding episodes. Following the initiation of emicizumab, six patients did not experience any further bleeding episodes. Mild episodes (epistaxis and muscular hematoma) were recorded in two cases, none of which required additional treatment.The 19-year-old patient with von Willebrand disease, who had a history of epistaxis and deep haematomas, showed a favourable response, with only one mild epistaxis episode during follow-up. No serious adverse effects were reported, with only one patient experiencing transient mild headache.Conclusion and Relevance Emicizumab proved to be effective and safe in this cohort of patients with haemophilia A, including those with inhibitors. Notably, the case of the patient with type III von Willebrand disease and an inhibitor demonstrated excellent tolerability and bleeding control. These findings support the potential role of emicizumab as a prophylactic treatment in bleeding disorders beyond haemophilia A.Conflict of Interest No conflict of interest",
  "authors": [
    {
      "affiliations": [
        "Hospital Universitario De Jaén, Farmacia Hospitalaria, Jaén, Spain"
      ],
      "name": "Á Moreno Lopez"
    },
    {
      "affiliations": [
        "Hospital Universitario De Jaén, Farmacia Hospitalaria, Jaén, Spain"
      ],
      "name": "B Morales Rivero"
    },
    {
      "affiliations": [
        "Hospital Universitario De Jaén, Farmacia Hospitalaria, Jaén, Spain"
      ],
      "name": "J Moreno Banegas"
    }
  ],
  "title": "6ER-034 Real-life experience with emicizumab: clinical outcomes and an exceptional case of type III von Willebrand disease with inhibitor",
  "uid": "7536c053-62ad-58f4-8e6c-93818ef65146"
}