Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
15 participants
OBSERVATIONAL
2019-12-18
2020-03-31
Brief Summary
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Detailed Description
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With the expansion of the prescription of eftrenonacog-alfa, the problem of accurately measuring its recovery has risen within hemostasis laboratories. Chronometric one-stage assays (OSA) are currently the most widely performed tests for FIX activity measurement. They are based on activated partial thromboplastin time (aPTT) reagent and factor-deficient plasma. Various aPTT reagents are commercialized. They could lead to a substantial variability in FIX results. Chromogenic two-stage assays (CSA) are less frequently used in clinical laboratories. They present fewer reagent choice compared to OSA. Several studies have evaluated OSA and CSA performances in samples spiked with eftrenonacog-alfa and have evidenced that some commonly used aPTT reagents would not be suitable for accurately monitoring this product. Whether these results are commutable to post-infusion samples collected from real-life patients remains unclear.
Unlike the measurement of a unique coagulation factor activity, thrombin generation assay (TGA) is a dynamic global test that explores the coagulation cascade as well as the anticoagulant pathways. TGA could be therefore a valuable tool to monitor replacement therapy in hemophilia B patients.
Hence the investigators sought in a real-life setting to compare 2 CSA and 1 OSA reagents to a product specific OSA in severe hemophilia B patients supplemented with eftrenonacog-alfa. The investigators also aimed to evaluate the pharmacodynamics (PD) of eftrenonacog-alfa using TGA.
Conditions
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Study Design
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COHORT
RETROSPECTIVE
Study Groups
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Single-group study
Severe haemophilia B patients substituted with eftrenonacog-alfa
Non interventional study
Non interventional study
Interventions
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Non interventional study
Non interventional study
Eligibility Criteria
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Inclusion Criteria
* treated with eftrenonacog-alfa
Exclusion Criteria
18 Years
ALL
No
Sponsors
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URC-CIC Paris Descartes Necker Cochin
OTHER
Assistance Publique - Hôpitaux de Paris
OTHER
Responsible Party
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Locations
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Service d'hématologie biologique - Hôpital Cochin
Paris, , France
Countries
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References
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Peyvandi F, Garagiola I, Boscarino M, Ryan A, Hermans C, Makris M. Real-life experience in switching to new extended half-life products at European haemophilia centres. Haemophilia. 2019 Nov;25(6):946-952. doi: 10.1111/hae.13834. Epub 2019 Aug 16.
Sommer JM, Buyue Y, Bardan S, Peters RT, Jiang H, Kamphaus GD, Gray E, Pierce GF. Comparative field study: impact of laboratory assay variability on the assessment of recombinant factor IX Fc fusion protein (rFIXFc) activity. Thromb Haemost. 2014 Nov;112(5):932-40. doi: 10.1160/TH13-11-0971. Epub 2014 Aug 21.
Kitchen S, Tiefenbacher S, Gosselin R. Factor Activity Assays for Monitoring Extended Half-Life FVIII and Factor IX Replacement Therapies. Semin Thromb Hemost. 2017 Apr;43(3):331-337. doi: 10.1055/s-0037-1598058. Epub 2017 Mar 6.
Dargaud Y, Beguin S, Lienhart A, Al Dieri R, Trzeciak C, Bordet JC, Hemker HC, Negrier C. Evaluation of thrombin generating capacity in plasma from patients with haemophilia A and B. Thromb Haemost. 2005 Mar;93(3):475-80. doi: 10.1160/TH04-10-0706.
Bowyer AE, Shepherd MF, Kitchen S, Maclean RM, Makris M. Measurement of extended half-life recombinant factor IX products in clinical practice. Int J Lab Hematol. 2019 Apr;41(2):e46-e49. doi: 10.1111/ijlh.12953. Epub 2018 Dec 7. No abstract available.
Collins PW, Fischer K, Morfini M, Blanchette VS, Bjorkman S; International Prophylaxis Study Group Pharmacokinetics Expert Working Group. Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia. 2011 Jan;17(1):2-10. doi: 10.1111/j.1365-2516.2010.02370.x. Epub 2010 Aug 22.
Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6.
Atsou S, Furlan F, Duchemin J, Ellouze S, Sourdeau E, Launois A, Roussel-Robert V, Stieltjes N, Combe S, Fontenay M, Curis E, Jourdi G. Pharmacodynamics of eftrenonacog-alfa (rFIX-Fc) in severe hemophilia B patients: A real-life study. Eur J Pharmacol. 2021 Jan 15;891:173764. doi: 10.1016/j.ejphar.2020.173764. Epub 2020 Nov 27.
Other Identifiers
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APHP191124
Identifier Type: -
Identifier Source: org_study_id
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