Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment

Acta Haematol. 2022;145(1):72-77. doi: 10.1159/000517813. Epub 2021 Aug 5.

Abstract

Acquired thrombocytopenic thrombotic purpura (aTTP) is an autoantibody-mediated disease against the enzyme A Disintegrin and Metalloprotease domain with ThromboSpondin-1 type motif 13, which until now has been treated with plasma exchange (PEX) and corticosteroids. A 29-year-old female patient, who presented with aTTP in the context of pregnancy, has developed multiple relapses after treatment with PEX, corticosteroids, and rituximab. Recently, caplacizumab, a nanobody against von Willebrand factor, has been approved for the treatment of aTTP. In our patient, caplacizumab achieved better disease control, with a lower platelet count restoration time, days of PEX and hospitalization duration, as compared to standard therapy, reproducing the results of clinical trials. Caplacizumab represents a significant advance in the treatment of aTTP, especially in cases of recurrent relapses.

Keywords: Caplacizumab; Plasma exchange; Thrombocytopenic purpura.

Publication types

  • Case Reports
  • Clinical Trial

MeSH terms

  • Adult
  • Female
  • Humans
  • Plasma Exchange*
  • Pregnancy
  • Pregnancy Complications, Hematologic / blood
  • Pregnancy Complications, Hematologic / therapy*
  • Purpura, Thrombotic Thrombocytopenic / blood
  • Purpura, Thrombotic Thrombocytopenic / therapy*
  • Single-Domain Antibodies / administration & dosage*

Substances

  • Single-Domain Antibodies
  • caplacizumab

Supplementary concepts

  • Thrombotic thrombocytopenic purpura, acquired