Once-weekly prophylactic treatment vs. on-demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B

Haemophilia : the Official Journal of the World Federation of Hemophilia
K KavakliPablo Rendo

Abstract

Limited data are available on optimal prophylaxis regimens of factor IX (FIX) replacements for patients with haemophilia B. This multicentre, open-label study evaluated the efficacy and safety of once-weekly prophylaxis with nonacog alfa compared with on-demand treatment in adolescent and adult patients. Males aged 12-65 years with moderately severe to severe haemophilia B (FIX:C ≤ 2%) were eligible for enrolment. Patients received on-demand treatment for 26 weeks, followed by once-weekly prophylaxis of 100 IU kg(-1) for 52 weeks. The primary efficacy end point was the annualized bleeding rate (ABR). Secondary end points included response to on-demand treatment, the number of infusions used to treat bleeding events, and the incidence of less-than-expected therapeutic effect (LETE). FIX:C was measured on day 1 and at weeks 26 and 78. Mean (±SD) ABR was lower during prophylaxis vs. on-demand treatment [3.6 (±4.6) vs. 32.9 (±17.4) events, respectively; P < 0.0001]. The majority (88.4%) of bleeding events had excellent or good responses upon the first infusion; 82.1% of events responded to the first infusion. No incident of LETE occurred. No thrombotic events or FIX inhibitors were reported. Eight of 17 FIX:C approximately 1 week a...Continue Reading

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Citations

Jun 30, 2016·Haemophilia : the Official Journal of the World Federation of Hemophilia·A SuzukiJ M Korth-Bradley
May 19, 2017·Haemophilia : the Official Journal of the World Federation of Hemophilia·M ScottC R M Hay
Nov 28, 2017·Expert Opinion on Biological Therapy·Yvonne BrennanEmmanuel J Favaloro
Nov 15, 2017·Blood Coagulation & Fibrinolysis : an International Journal in Haemostasis and Thrombosis·Pablo RendoJoan M Korth-Bradley
Feb 23, 2020·Journal of Thrombosis and Haemostasis : JTH·Maria Elisa MancusoElena Santagostino
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Jun 30, 2019·BMC Bioinformatics·Lennon Meléndez-ArandaMarina María de Jesús Romero-Prado
Jul 10, 2019·Expert Review of Hematology·Miguel EscobarClaude Negrier
Dec 6, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Ri LiesnerAnnarita Tagliaferri
Mar 30, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·David M MannDarrel W Stafford
Feb 26, 2021·Blood Coagulation & Fibrinolysis : an International Journal in Haemostasis and Thrombosis·Bartholomew J TortellaKaan Kavakli
Jul 7, 2021·Expert Opinion on Biological Therapy·María Teresa Álvarez RománVictor Jiménez-Yuste

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