{
  "abstract": "Background and Aims. Joint damage and impaired quality of life (QoL) remain major long-term complications in patients with hemophilia A (HA). Real-world longitudinal data after switching to emicizumab prophylaxis are limited. The aim of our study is to evaluate longitudinal changes in joint health and QoL in moderate/severe HA patients switching to emicizumab. Methods. Among 101 patients switching from FVIII to emicizumab at our center, 41 underwent systematic joint health assessment (between 2020 and 2024), at baseline, 1 year after the switch, and after 2 years (n=21). Joint health was assessed using the Hemophilia Joint Health Score (HJHS) and the Hemophilic Early Arthropathy Detection with Ultrasound (HEAD-US) score, the QoL with the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) questionnaire, and the EuroQol Visual Analogue Scale (EQ-VAS). The Anualized Bleeding Ratio (ABR) was recorded. Changes between −1 and +1 points in HEAD-US were considered stable; ≥2-point variation indicated improvement or worsening. Continuous variables are reported as median (IQR/range). Fisher’s exact test compared worsening in target vs. non-target joints. QoL changes were analyzed with Wilcoxon and Friedman tests (p<0.05). Results. The HEAD-US score remained mostly stable from baseline (median 12, IQR 3-19) to 1 (14, IQR 4-20) and 2 years (11, IQR 2-18). For HJHS, 32/41 patients (78.0%) were stable or improved at 1 year, and 15/21 (73.7%) showed stability or improvement at 2 years. EQ-VAS remained stable. EQ-5D-5L showed significant improvements in pain/discomfort (p=0.024) and mobility (p=0.030) (Figure). To provide a joint-level perspective, 224 joints were evaluated at 1 year and 106 at 2 years, with stability in HEAD-US score predominating (>95% unchanged at 1 year and similarly high rates at 2 years). At 1 year, worsening occurred only in 6/103 target joints compared with 1/121 non-target joints (OR 7.42, p=0.050); at 2 years, worsening was observed in 3/39 target joints versus 1/67 non-target joints (OR 5.5, p=0.14). From baseline to 1 year, patients with an ABR of 0 increased (16/41 vs 22/41; risk difference RD 15%), whereas the proportion of patients with ABR ≥ 3 decreased (10/41 vs 5/41; RD 12%). Conclusions. In our cohort of HA patients switching to emicizumab, joint health was preserved. Most joints remained stable, with limited worsening mainly involving target joints. Improvements in pain/discomfort and mobility further support the clinical benefit of emicizumab beyond structural outcomes.",
  "abstract_shown": true,
  "affiliations": [],
  "article_type": null,
  "arxiv": null,
  "author_total": 1,
  "authors": [
    {
      "name": "Sara Ceglie"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/8f1kenv11vjb0.bib",
  "cited_by": 0,
  "doi": null,
  "fulltext_url": "https://www.btvb.org/btvb/article/view/704",
  "id": "8f1kenv11vjb0",
  "issns": [
    "2785-5309"
  ],
  "landing_url": "https://www.btvb.org/btvb/article/view/704",
  "language": "en",
  "licence": "CC-BY-NC",
  "licence_label": "CC BY-NC",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by-nc/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC666-701"
    ]
  ],
  "openalex": null,
  "pdf_url": null,
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-26",
  "publisher": "PAGEPress Publications",
  "ris": "https://research.draconitic.com/paper/8f1kenv11vjb0.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:47:26Z",
      "native_id": "1d6f27103e034ff59fdb92174a4302d6",
      "source": "doaj",
      "url": "https://www.btvb.org/btvb/article/view/704"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "lcc:RC666-701",
      "subfield": "cardiovascular"
    }
  ],
  "title": "CO002 | RETROSPECTIVE LONGITUDINAL ASSESSMENT OF JOINT HEALTH AND QUALITY OF LIFE IN ADOLESCENT AND ADULT PATIENTS AFFECTED BY MODERATE OR SEVERE HAEMOPHILIA A WITH OR WITHOUT INHIBITOR SWITCHING FROM INTRAVENOUS FVIII TREATMENT TO SUBCUTANEOUS PROPHYLAXIS WITH EMICIZUMAB",
  "title_text": "CO002 | RETROSPECTIVE LONGITUDINAL ASSESSMENT OF JOINT HEALTH AND QUALITY OF LIFE IN ADOLESCENT AND ADULT PATIENTS AFFECTED BY MODERATE OR SEVERE HAEMOPHILIA A WITH OR WITHOUT INHIBITOR SWITCHING FROM INTRAVENOUS FVIII TREATMENT TO SUBCUTANEOUS PROPHYLAXIS WITH EMICIZUMAB",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:47:26Z",
  "url": "https://research.draconitic.com/paper/8f1kenv11vjb0",
  "venue": "Bleeding, Thrombosis and Vascular Biology",
  "versions": []
}