{
  "abstract": "Background: Concizumab is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody approved in the US, Europe and other countries for once-daily, subcutaneous prophylaxis for patients aged ≥12 years with haemophilia A/B with or without inhibitors. Aim: To assess concizumab efficacy and safety in paediatric participants with haemophilia A/B with inhibitors (HAwI/HBwI) at the explorer10 (NCT05135559) 32-week cut-off. Methods: Explorer10 is a multi-centre, non-randomised phase 3 study of male paediatric partici-pants (<12 years) who were concizumab-naïve (arm 1) or previously treated with concizumab via compassionate use (arm 2). Concizumab-naïve participants received a 1.0 mg/kg loading dose (Day 1), followed by 0.20 mg/kg daily from Day 2, with potential dose adjustment at 5–8 weeks to 0.15 or 0.25 mg/kg based on plasma concentration. The primary endpoint was the number of treated spontaneous/traumatic bleeding episodes during concizumab prophylaxis vs previous on-demand treatment (≥26 weeks; historical records) in arm 1. Cut-off was when all arm 1 par-ticipants completed week 32 or withdrew. Safety and PK/PD parameters were also evaluated. Informed consent was obtained and the study was approved by local ethics committees. Results: In 24 participants, estimated mean ABR (95% CI) was 2.08 (1.27; 3.41) on concizumab prophylaxis vs 11.51 (7.75; 17.09) with previous on-demand treatment. The ABR ratio was 0.18 (0.11; 0.29; P < 0.001), representing an 82% ABR reduction and demonstrating superiority of con-cizumab prophylaxis. Concizumab plasma concentration remained stable over time, free TFPI levels decreased and remained stable, and the geometric mean thrombin peak increased to within the normal adult range. Concizumab was well tolerated, with no safety concerns identi-fied. Conclusion: In paediatric HAwI/HBwI, concizumab prophylaxis was effective in bleed prevention compared with previous observations.",
  "abstract_shown": true,
  "affiliations": [
    "Vilnius University, Lithuania",
    "Feinberg School of Medicine, United States",
    "McMaster Children's Hospital, Canada",
    "Novo Nordisk, Denmark",
    "La Paz University Hospital-IdiPAz, Spain",
    "Ege University Faculty of Medicine, Türkiye",
    "University of the Witwatersrand and National Health Laboratory Service, South Africa",
    "Great Ormond Street Hospital for Children NHS Foundation Trust, United Kingdom",
    "St Marianna University School of Medicine, Japan",
    "University Hospital Medical Centre Beni Messous, Algeria",
    "Post Graduate Institute of Child Health, India",
    "University of Washington School of Medicine, United States",
    "Careggi University Hospital, Italy",
    "Novo Nordisk, Rio de Janeiro, Brazil"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 15,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Sonata Šaulytė TRAKYMIEN"
    },
    {
      "affs": [
        1
      ],
      "name": "Rukhmi BHAT"
    },
    {
      "affs": [
        2
      ],
      "name": "Anthony Kc CHAN"
    },
    {
      "affs": [
        3
      ],
      "name": "Anastasios GIAKOUMIS"
    },
    {
      "affs": [
        4
      ],
      "name": "Victor Jiménez YUSTE"
    },
    {
      "affs": [
        5
      ],
      "name": "Kaan KAVAKLI"
    },
    {
      "affs": [
        3
      ],
      "name": "Maria KNUDSEN"
    },
    {
      "affs": [
        6
      ],
      "name": "Johnny MAHLANGU"
    },
    {
      "affs": [
        7
      ],
      "name": "Mary MATHIAS"
    },
    {
      "affs": [
        8
      ],
      "name": "Chiai NAGAE"
    },
    {
      "affs": [
        9
      ],
      "name": "Salim Mohamed NEKKAL"
    },
    {
      "affs": [
        10
      ],
      "name": "Nita RADHAKRISHNAN"
    },
    {
      "affs": [
        11
      ],
      "name": "Allison P WHEELER"
    },
    {
      "affs": [
        12
      ],
      "name": "Giancarlo CASTAMAN"
    },
    {
      "affs": [
        13
      ],
      "name": "Vanessa Maira Lima CAMPOS"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/ejd4h0vvjp38z.bib",
  "cited_by": 0,
  "doi": "10.1016/j.htct.2026.107996",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926017438",
  "id": "ejd4h0vvjp38z",
  "issns": [
    "2531-1379",
    "2531-1387"
  ],
  "landing_url": "https://doi.org/10.1016/j.htct.2026.107996",
  "language": "en",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC633-647.5"
    ],
    [
      "openalex",
      "2720|Hemophilia Treatment and Research"
    ]
  ],
  "openalex": "W7223104074",
  "pdf_url": "https://www.sciencedirect.com/science/article/pii/S2531137926017438/pdf",
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/ejd4h0vvjp38z.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:43:19Z",
      "native_id": "6323a3db6c3f4bf18a8b6fe418b1bb12",
      "source": "doaj",
      "url": "https://doi.org/10.1016/j.htct.2026.107996"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "lcc:RC633-647.5",
      "subfield": "endocrinology-metabolism"
    }
  ],
  "title": "CONCIZUMAB PROPHYLAXIS IN PAEDIATRIC PARTICIPANTS WITH HAEMOPHILIA A/B WITH INHIBITORS IN THE PHASE 3 EXPLORER10 STUDY: EFFICACY, SAFETY AND PK/PD RESULTS FROM THE 32-WEEK CUT-OFF",
  "title_text": "CONCIZUMAB PROPHYLAXIS IN PAEDIATRIC PARTICIPANTS WITH HAEMOPHILIA A/B WITH INHIBITORS IN THE PHASE 3 EXPLORER10 STUDY: EFFICACY, SAFETY AND PK/PD RESULTS FROM THE 32-WEEK CUT-OFF",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:43:19Z",
  "url": "https://research.draconitic.com/paper/ejd4h0vvjp38z",
  "venue": "Hematology, Transfusion and Cell Therapy",
  "versions": []
}