{
  "abstract": "Caplacizumab, an anti–von Willebrand factor nanobody, is currently indicated for the treatment of patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in combination with therapeutic plasma exchange and immunosuppression. This retrospective chart review assessed the safety and effectiveness of caplacizumab in patients with iTTP aged <18 years in 3 countries. Overall, 30 patients (median age at diagnosis: 14 years [range, 1-17]) were enrolled. Medical data spanning from the date of iTTP diagnosis to the earliest date of chart extraction initiation were collected retrospectively. Clinical response was achieved by 29 of 30 patients (96.7%) and clinical remission by 24 of 30 patients (80%). The median time to platelet count response was 5 days (95% confidence interval, 3-6). Refractory disease and relapse were each reported in 2 patients (6.7%), with no exacerbations. Overall, 24 of 27 evaluable patients achieved ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1, motif member 13) activity of ≥20% during the observation period. Fifteen patients (50%) experienced adverse events (AEs), with 9 serious AEs reported; 8 patients (26.7%) reported bleeding events, mainly epistaxis. One death occurred, which was deemed unrelated to the treatment. Caplacizumab treatment was effective and well tolerated in this pediatric population, with no new safety signals, supporting its use in pediatric patients aged <18 years with iTTP.",
  "abstract_shown": true,
  "affiliations": [
    "Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Atlanta, GA; Department of Pediatrics, Emory University School of Medicine, Atlanta, GA; Correspondence: Satheesh Chonat, Pediatrics, Emory University School of Medicine, 2015 Uppergate Dr, Atlanta, GA 30322;",
    "Department of Hematology, Reference Center for Thrombotic Microangiopathies, Saint-Antoine University Hospital, Assistance publique–hôpitaux de Paris, Paris, France",
    "Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Texas Children’s Cancer and Hematology Center, Houston, TX",
    "Pediatric Nephrology, Maladies Rénales Héréditaires de l'Enfant et de l'Adulte Reference Center, Reference Center for Thrombotic Microangiopathies, Necker-Enfants Malades Hospital, Assistance publique–hôpitaux de Paris, Imagine Institute, INSERM U1163, Paris Cité University, Paris, France",
    "National Reference Center for Thrombotic Microangiopathies, Paris, France; Department of Nephrology, Dialysis and Transplantation, University of Lille, Lille University Hospital, Lille, France",
    "Department of Pediatric Nephrology, Reference Center for Rare Kidney Diseases Nephrogones, Rare Disease Networks Orphan Kidney Diseases and European Rare Kidney Disease Reference Network, University Hospital of Lyon, Bron, France",
    "Great Ormond Street Hospital for Children Foundation Trust, London, United Kingdom",
    "Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA",
    "Division of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, OH",
    "Sanofi, Cambridge, MA",
    "Sanofi, Morristown, NJ",
    "University College London Hospitals NHS Foundation Trust, London, United Kingdom"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 13,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Satheesh Chonat"
    },
    {
      "affs": [
        1
      ],
      "name": "Paul Coppo"
    },
    {
      "affs": [
        2
      ],
      "name": "Clay Cohen"
    },
    {
      "affs": [
        3
      ],
      "name": "Olivia Boyer"
    },
    {
      "affs": [
        4
      ],
      "name": "Francois Provot"
    },
    {
      "affs": [
        5
      ],
      "name": "Anne-Laure Sellier-Leclerc"
    },
    {
      "affs": [
        6
      ],
      "name": "Alice Taylor"
    },
    {
      "affs": [
        7
      ],
      "name": "Michele P. Lambert"
    },
    {
      "affs": [
        8
      ],
      "name": "Spero Cataland"
    },
    {
      "affs": [
        9
      ],
      "name": "Ana Paula Marques"
    },
    {
      "affs": [
        9
      ],
      "name": "Meredith C. Foster"
    },
    {
      "affs": [
        10
      ],
      "name": "Sriya Gunawardena"
    },
    {
      "affs": [
        11
      ],
      "name": "Marie Scully"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/01csk0xafh5br.bib",
  "cited_by": 1,
  "doi": "10.1016/j.bvth.2026.100224",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2950327226000896",
  "id": "01csk0xafh5br",
  "issns": [
    "2950-3272"
  ],
  "landing_url": "https://doi.org/10.1016/j.bvth.2026.100224",
  "language": "en",
  "licence": "CC-BY-NC-ND",
  "licence_label": "CC BY-NC-ND",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC633-647.5"
    ],
    [
      "openalex",
      "2403|Complement system in diseases"
    ]
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  "openalex": "W7214329925",
  "pdf_url": "https://www.sciencedirect.com/science/article/pii/S2950327226000896/pdf",
  "pmcid": null,
  "pmid": null,
  "published": "2026-11-06",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/01csk0xafh5br.ris",
  "sources": [
    {
      "first_seen": "2026-11-06T14:21:05Z",
      "native_id": "04eb3bf173974f53836f727f4bf4cda3",
      "source": "doaj",
      "url": "https://doi.org/10.1016/j.bvth.2026.100224"
    }
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  "subjects": [
    {
      "field": "biology",
      "origin": "openalex:2403|Complement system in diseases",
      "subfield": "immunology"
    }
  ],
  "title": "Safety and effectiveness of caplacizumab in pediatric patients with iTTP: real-world experience",
  "title_text": "Safety and effectiveness of caplacizumab in pediatric patients with iTTP: real-world experience",
  "type": "peer-reviewed",
  "updated_at": "2026-11-07T14:20:51Z",
  "url": "https://research.draconitic.com/paper/01csk0xafh5br",
  "venue": "Blood Vessels, Thrombosis & Hemostasis",
  "versions": []
}