[Factor IX assays in treated hemophilia B patients]

Ann Biol Clin (Paris). 2019 Feb 1;77(1):41-52. doi: 10.1684/abc.2019.1414.
[Article in French]

Abstract

Replacement therapy with plasma-derived or recombinant FIX (pdFIX or rFIX) concentrates is the standard of treatment in patients with hemophilia B. The method predominantly used for measuring factor IX (FIX:C) levels is the one-stage clotting assay (OSA) but this method depends on the activated partial thromboplastin time (APTT) reagent and the coagulation analyzer used, and wide variations in the measurements of FIX recovery have been reported with some factor concentrates. The French study group on the biology of hemorrhagic diseases (a collaborative group of the GFHT and MHEMO network), presents a review of the literature and proposals for the monitoring of FIX:C levels in treated hemophilia B patients. The use of OSA calibrated with a plasma reference tested against the current FIX WHO International Standard is recommended for the monitoring of patients treated with pdFIX or rFIX. Chromogenic substrate assays (CSA) are adequate for the monitoring of patients treated with Rixubis®, but data available for Benefix® are currently too limited. For extended half-life rFIX (EHL-rFIX), large discordances in the FIX:C levels measured were evidenced, depending on the method and reagents used. Great attention is therefore required for measuring FIX:C levels by OSA in patients substituted by EHL-rFIX. Commercial kits for CSA are not equivalent, and although potentially useful, they are not validated for all EHL-rFIX. Most of recent studies reported data obtained with spiked plasmas, which deserve to be confirmed on plasma samples collected in treated patients.

Keywords: chromogenic assays; chronometric assays; factor IX; hemophilia B.

Publication types

  • Review

MeSH terms

  • Blood Chemical Analysis / methods
  • Blood Coagulation Tests / methods
  • Factor IX / analysis*
  • Hemophilia B / blood*
  • Hemophilia B / diagnosis*
  • Hemophilia B / therapy
  • Humans
  • Monitoring, Physiologic / methods*
  • Prognosis

Substances

  • Factor IX