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

Introduction Limited data are available on optimal prophylaxis regimens of factor IX (FIX) replacements for patients with haemophilia B. Aim This multicentre, open‐label study evaluated the efficacy and safety of once‐weekly prophylaxis with nonacog alfa compared with on‐demand treatment in adolesce...

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Veröffentlicht in:Haemophilia : the official journal of the World Federation of Hemophilia Jg. 22; H. 3; S. 381 - 388
Hauptverfasser: Kavakli, K., Smith, L., Kuliczkowski, K., Korth-Bradley, J., You, C. W., Fuiman, J., Zupančić-Šalek, S., Abdul Karim, F., Rendo, P.
Format: Journal Article
Sprache:Englisch
Veröffentlicht: England Blackwell Publishing Ltd 01.05.2016
Wiley Subscription Services, Inc
Schlagworte:
Adolescent
Adult
Aged
BeneFIX
Bleeding
Child
Coagulants - adverse effects
Coagulants - therapeutic use
Coagulation factors
Dosage
Drug Administration Schedule
factor IX
Factor IX - genetics
Factor IX - metabolism
Factor IX - therapeutic use
Factor IX deficiency
haemophilia B
Hemophilia
Hemophilia B - drug therapy
Hemorrhage - epidemiology
Hemorrhage - prevention & control
Humans
Male
Middle Aged
pharmacokinetics
prevention
Prophylaxis
Recombinant Proteins - biosynthesis
Recombinant Proteins - genetics
Recombinant Proteins - therapeutic use
safety
Thrombosis - etiology
Treatment Outcome
Young Adult
pharmacokinetics
factor IX
haemophilia B
BeneFIX
safety
prevention
ISSN:1351-8216, 1365-2516, 1365-2516
Online-Zugang:Volltext
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Zusammenfassung:Introduction Limited data are available on optimal prophylaxis regimens of factor IX (FIX) replacements for patients with haemophilia B. Aim 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. Methods 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. Results 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 after dosing were >2 IU dL−1 (min–max of 2.13–10.39 IU dL−1). Conclusions Once‐weekly prophylaxis of 100 IU kg−1 was associated with lower ABR compared with on‐demand treatment in adolescents and adults with moderately severe to severe haemophilia B. Once‐weekly prophylaxis was well tolerated, with a similar safety profile as that reported during the on‐demand treatment period. Residual FIX:C may be supportive of effectiveness.
Bibliographie:ArticleID:HAE12878
istex:F00032E01C8C9F4BA63DCB213B0F0BDB773E0D6C
Pfizer
ark:/67375/WNG-XBMQFMGD-X
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ISSN:1351-8216
1365-2516
1365-2516
DOI:10.1111/hae.12878