Immune-mediated thrombotic thrombocytopenic purpura in childhood treated by caplacizumab, about 3 cases

J Nephrol. 2022 Mar;35(2):653-656. doi: 10.1007/s40620-021-00992-5. Epub 2021 Feb 22.


Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare disease in adults and exceptional in childhood. Caplacizumab has proven its effectiveness in the treatment of iTTP in adulthood in association with standard of care. Unfortunately, this treatment is restricted to adults. We report our experience in three children who were treated successfully with caplacizumab.

MeSH terms

  • Adult
  • Child
  • Humans
  • Plasma Exchange
  • Purpura, Thrombotic Thrombocytopenic* / diagnosis
  • Purpura, Thrombotic Thrombocytopenic* / drug therapy
  • Rare Diseases / therapy
  • Single-Domain Antibodies* / therapeutic use


  • Single-Domain Antibodies
  • caplacizumab