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Peer-reviewedCC BY-NC-NDBlood Vessels, Thrombosis & Hemostasis

Safety and effectiveness of caplacizumab in pediatric patients with iTTP: real-world experience

  • Satheesh Chonat1
  • Paul Coppo2
  • Clay Cohen3
  • Olivia Boyer4
  • Francois Provot5
  • Anne-Laure Sellier-Leclerc6
  • Alice Taylor7
  • Michele P. Lambert8
  • Spero Cataland9
  • Ana Paula Marques10
  • Meredith C. Foster10
  • Sriya Gunawardena11
1 more authors
  • Marie Scully12
12 affiliations
  1. 1Aflac 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;
  2. 2Department of Hematology, Reference Center for Thrombotic Microangiopathies, Saint-Antoine University Hospital, Assistance publique–hôpitaux de Paris, Paris, France
  3. 3Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Texas Children’s Cancer and Hematology Center, Houston, TX
  4. 4Pediatric 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
  5. 5National Reference Center for Thrombotic Microangiopathies, Paris, France; Department of Nephrology, Dialysis and Transplantation, University of Lille, Lille University Hospital, Lille, France
  6. 6Department 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
  7. 7Great Ormond Street Hospital for Children Foundation Trust, London, United Kingdom
  8. 8Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
  9. 9Division of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, OH
  10. 10Sanofi, Cambridge, MA
  11. 11Sanofi, Morristown, NJ
  12. 12University College London Hospitals NHS Foundation Trust, London, United Kingdom
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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.

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Cite

BibTeXRIS
BibTeX
@article{chonat2026safety,
  title = {{Safety and effectiveness of caplacizumab in pediatric patients with iTTP: real-world experience}},
  author = {Satheesh Chonat and Paul Coppo and Clay Cohen and Olivia Boyer and Francois Provot and Anne-Laure Sellier-Leclerc and Alice Taylor and Michele P. Lambert and Spero Cataland and Ana Paula Marques and Meredith C. Foster and Sriya Gunawardena and Marie Scully},
  journal = {Blood Vessels, Thrombosis \& Hemostasis},
  year = {2026},
  month = nov,
  doi = {10.1016/j.bvth.2026.100224},
  url = {https://doi.org/10.1016/j.bvth.2026.100224},
  note = {CC BY-NC-ND},
}
RIS
TY  - JOUR
TI  - Safety and effectiveness of caplacizumab in pediatric patients with iTTP: real-world experience
AU  - Satheesh Chonat
AU  - Paul Coppo
AU  - Clay Cohen
AU  - Olivia Boyer
AU  - Francois Provot
AU  - Anne-Laure Sellier-Leclerc
AU  - Alice Taylor
AU  - Michele P. Lambert
AU  - Spero Cataland
AU  - Ana Paula Marques
AU  - Meredith C. Foster
AU  - Sriya Gunawardena
AU  - Marie Scully
PY  - 2026
DA  - 2026/11/06
JO  - Blood Vessels, Thrombosis & Hemostasis
DO  - 10.1016/j.bvth.2026.100224
UR  - https://doi.org/10.1016/j.bvth.2026.100224
AB  - 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.
SN  - 2950-3272
ER  -