In silico evaluation of limited sampling strategies for individualized dosing of extended half-life factor IX concentrates in hemophilia B patients.

Purpose: Hemophilia B is a bleeding disorder, caused by a factor IX (FIX) deficiency. Recently, FIX concentrates with extended half-life (EHL) have become available. Prophylactic dosing of EHL-FIX concentrates can be optimized by assessment of individual pharmacokinetic (PK) parameters. To determine...

Full description

Bibliographic Details
Published in:European Journal of Clinical Pharmacology Vol. 78; no. 2; pp. 237 - 250
Main Authors: Preijers, T., van Spengler, M. W. F., Meijer, K., Fijnvandraat, K., Fischer, K., Leebeek, F. W. G., Cnossen, M. H., Mathôt, R. A. A.
Format: equations & formulas research tables/charts Journal Article
Published: Springer Nature Feb2022
Online Access:View this record in EBSCOhost
fields @attributes:
  recordID: 1
pdfLink:
plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=154581363&site=ehost-live
header:
  @attributes:
    shortDbName: ccm
    uiTerm: 154581363
    longDbName: CINAHL Complete
    uiTag: AN
  controlInfo:
    bkinfo:
    dissinfo:
    jinfo:
      jid:
        00316970
        NP9
      jtl: European Journal of Clinical Pharmacology
      issn: 00316970
      maglogo: N
    pubinfo:
      dt: Feb2022
      vid: 78
      iid: 2
      pid: 237
      pub: Springer Nature
      place: New York, New York
    artinfo:
      ui:
        154581363
        152996770
        154581363
        154581363
        10.1007/s00228-021-03173-2
        154581363
      ppf: 237
      ppct: 13
      formats:
        fmt:
          – @attributes:
              type: T
          – @attributes:
              type: P
      tig:
        atl: In silico evaluation of limited sampling strategies for individualized dosing of extended half-life factor IX concentrates in hemophilia B patients.
      aug:
        au:
          Preijers, T.
          van Spengler, M. W. F.
          Meijer, K.
          Fijnvandraat, K.
          Fischer, K.
          Leebeek, F. W. G.
          Cnossen, M. H.
          Mathôt, R. A. A.
        affil: Hospital Pharmacy-Clinical Pharmacology, Amsterdam University Medical Center, Amsterdam, The Netherlands
      sug:
        subj:
          Hemophilia B Drug Therapy
          Sampling Methods Evaluation
          Biological Markers
          Human
          Simulations
          Descriptive Statistics
      ab: Purpose: Hemophilia B is a bleeding disorder, caused by a factor IX (FIX) deficiency. Recently, FIX concentrates with extended half-life (EHL) have become available. Prophylactic dosing of EHL-FIX concentrates can be optimized by assessment of individual pharmacokinetic (PK) parameters. To determine these parameters, limited sampling strategies (LSSs) may be applied. The study aims to establish adequate LSSs for estimating individual PK parameters of EHL-FIX concentrates using in silico evaluation. Methods: Monte Carlo simulations were performed to obtain FIX activity versus time profiles using published population PK models for N9-GP (Refixia), rFIXFc (Alprolix), and rIX-FP (Idelvion). Fourteen LSSs, containing three or four samples taken within 8 days after administration, were formulated. Bayesian analysis was applied to obtain estimates for clearance (CL), half-life (t1/2), time to 1% (Time1%), and calculated weekly dose (Dose1%). Bias and precision of these estimates were assessed to determine which LSS was adequate. Results: For all PK parameters of N9-GP, rFIXFc and rIX-FP bias was generally acceptable (range: −5% to 5%). For N9-GP, precision of all parameters for all LSSs was acceptable (< 25%). For rFIXFc, precision was acceptable for CL and Time1%, except for t1/2 (range: 27.1% to 44.7%) and Dose1% (range: 12% to 29.4%). For rIX-FP, all LSSs showed acceptable bias and precision, except for Dose1% using LSS with the last sample taken on day 3 (LSS 6 and 10). Conclusion: Best performing LSSs were LSS with samples taken at days 1, 5, 7, and 8 (N9-GP and rFIXFc) and at days 1, 4, 6, and 8 (rIX-FP), respectively.
      pubtype: Academic Journal
      doctype:
        equations & formulas
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
    refInfo:
    holdings:
      @attributes:
        islocal: N