Article Text

Download PDFPDF
Patient-centered approach to managing factor XIII deficiency
  1. Varun Iyengar1,
  2. Caitlin Montcrieff2 and
  3. Salley Pels2
  1. 1Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA
  2. 2Hasbro Children's Hospital, Providence, Rhode Island, USA
  1. Correspondence to Dr Varun Iyengar; viyengar{at}bidmc.harvard.edu

Abstract

Factor XIII (FXIII) is a thrombin-activated protransglutaminase that plays a key role in blood clot formation. Congenital FXIII A-subunit deficiency represents a rare bleeding disorder that affects one in 2–3 million individuals worldwide and is treated with recombinant FXIII (rFXIII). However, due to the rarity of the disease, clinicians are often left to weigh individual variation in FXIII activity and/or symptoms to optimally guide dosing. Cases often become further complicated when patients experience refractory bleeding, which can be difficult to treat. This report describes an approach to rFXIII dosing in two patients who required deviation from standard protocols to maintain therapeutic FXIII troughs. We highlight limitations in our understanding of FXIII deficiency management, while also providing an example of the application of pharmacokinetic data to individualise therapy for improved outcomes. Finally, the case reminds us of the importance of patient-centered, cost-conscious care and multidisplinary teamwork in complex cases.

  • haematology (drugs and medicines)
  • haematology (incl blood transfusion)

Statistics from Altmetric.com

Footnotes

  • Contributors VI contributed to the design and implementation of the research, to the analysis of the results, and to the writing of the manuscript. CM and SP developed the theoretical framework and supervised the project.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Provenance and peer review Not commissioned; externally peer reviewed.

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.