Should we consider caplacizumab as routine treatment for acute thrombotic thrombocytopenic purpura? An expert perspective on the pros and cons

Expert Rev Hematol. 2024 Jan-Mar;17(1-3):9-25. doi: 10.1080/17474086.2024.2318347. Epub 2024 Feb 20.

Abstract

Introduction: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening disorder. Caplacizumab has been the latest drug incorporated into the initial treatment of acute episodes, allowing for faster platelet recovery and a decrease in refractoriness, exacerbation, thromboembolic events, and mortality. However, caplacizumab is also associated with a bleeding risk and higher treatment costs, which prevent many centers from using it universally.

Areas covered: Studies that included iTTP and/or caplacizumab to date were selected for this review using PubMed and MEDLINE platforms. We describe outcomes in the pre-caplacizumab era and after it, highlighting the benefits and risks of its use early in frontline, and also pointing out special situations that require careful management.

Expert opinion: It is clear that the availability of caplacizumab has significantly and favorably impacted the management of iTTP patients. Whether this improvement is cost-effective still remains uncertain, and data on long-term sequelae and different healthcare systems will help to clarify this point. In addition, evidence of the bleeding/thrombotic risk of iTTP patients under this drug needs to be better addressed in future studies.

Keywords: ADAMTS13; Caplacizumab; iTTP; outcome; therapy; thrombotic thrombocytopenic purpura.

Publication types

  • Review

MeSH terms

  • ADAMTS13 Protein / therapeutic use
  • Humans
  • Plasma Exchange
  • Purpura, Thrombocytopenic, Idiopathic* / therapy
  • Purpura, Thrombotic Thrombocytopenic* / drug therapy
  • Single-Domain Antibodies*
  • von Willebrand Factor

Substances

  • caplacizumab
  • von Willebrand Factor
  • ADAMTS13 Protein
  • Single-Domain Antibodies