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Tytuł pozycji:

Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset.

Tytuł :
Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset.
Autorzy :
Tortella BJ; Pfizer Inc., Collegeville, Pennsylvania, USA.
Carr ME; Pfizer Inc., Collegeville, Pennsylvania, USA.
Rendo P; Pfizer Inc., Collegeville, Pennsylvania, USA.
Korth-Bradley J; Pfizer Inc., Collegeville, Pennsylvania, USA.
Smith LM; Pfizer Inc., Collegeville, Pennsylvania, USA.
Kavakli K; Department of Pediatric Hematology, University of Ege, Izmir, Turkey.
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Źródło :
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis [Blood Coagul Fibrinolysis] 2021 Apr 01; Vol. 32 (3), pp. 180-185.
Typ publikacji :
Journal Article
Język :
English
Imprint Name(s) :
Publication: London : Lippincott Williams And Wilkins
Original Publication: Oxford, UK : Rapid Communications of Oxford Ltd., c1990-
References :
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Iorio A, Fischer K, Blanchette V, Rangarajan S, Young G, Morfini M. Tailoring treatment of haemophilia B: accounting for the distribution and clearance of standard and extended half-life FIX concentrates. Thromb Haemost 2017; 117:1023–1030.
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Gui T, Lin HF, Jin DY, Hoffman M, Straight DL, Roberts HR, et al. Circulating and binding characteristics of wild-type factor IX and certain Gla domain mutants in vivo. Blood 2002; 100:153–158.
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Valentino LA, Rusen L, Elezovic I, Smith LM, Korth-Bradley JM, Rendo P. Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects. Haemophilia 2014; 20:398–406.
Kavakli K, Smith L, Kuliczkowski K, Korth-Bradley J, You CW, Fuiman J, et al. Once-weekly prophylactic treatment versus on-demand treatment with nonacog alfa in patients with moderately severe to severe hemophilia B. Haemophilia 2016; 22:381–388.
Bjorkman S, Carlsson M, Berntorp E. Pharmacokinetics of factor IX in patients with haemophilia B. Methodological aspects and physiological interpretation. Eur J Clin Pharmacol 1994; 46:325–332.
Rendo P, Shafer F, Korth-Bradley JM, Sivamurthy K, Korin J. Factors that influence the bleeding phenotype in severe hemophilic patients. Blood Coagul Fibrinolysis 2013; 24:683–690.
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Feng D, Stafford KA, Broze GJ, Stafford DW. Evidence of clinically significant extravascular stores of factor IX. J Thromb Haemost 2013; 11:2176–2178.
Chang P, Aronson DL, Borenstein DG, Kessler CM. Coagulant proteins and thrombin generation in synovial fluid: a model for extravascular coagulation. Am J Hematol 1995; 50:79–83.
Sun J, Hakobyan N, Valentino LA, Feldman BL, Samulski RJ, Monahan PE. Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX. Blood 2008; 112:4532–4541.
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Korth-Bradley JM, Valentino LA, Rendo P, Shafer FE, Smith L, Baumann JA, et al. Pharmacokinetic/pharmacodynamic assessment of reformulated recombinant coagulation factor IX in adults and children with severe hemophilia B. Poster presented at the Annual Meeting of the American Society of Hematology, San Diego, California, USA. 2011.
Molecular Sequence :
ClinicalTrials.gov NCT01335061
Entry Date(s) :
Date Created: 20210225 Latest Revision: 20210423
Update Code :
20210423
DOI :
10.1097/MBC.0000000000001012
PMID :
33631774
Czasopismo naukowe
In a pivotal, multicenter, open-label study, 25 patients aged 12-54 years with moderately severe/severe hemophilia B received on-demand nonacog alfa (6 months; dose at investigator's discretion) followed by once-weekly prophylaxis with nonacog alfa 100 IU/kg (12 months). During prophylaxis, patients had a median spontaneous annualized bleeding rate (sABR) of 1.0 and significant reductions in ABR (P < 0.0001). This post hoc analysis examined the time of onset of spontaneous bleeding events (sBEs) and spontaneous target joint bleeding events (sTJBEs). The postdosing day (D) of onset of sBEs observed during prophylaxis and steady-state FIX activity data (FIX:C) between 144 and 196 h postdose were collected at weeks 26 and 78. Twelve patients (48%) had no sBEs; the remaining 13 (52%) had the following onset of sBEs: less than 1 D (0%), 1 to less than 2D (5%), 2 to less than 3 D (22%), 3 to less than 4 D (9%), 4 to less than 5D (22%), 5 to less than 6D (23%), 6 to less than 7D (11%), and at least 7D (8%). Reductions in sBEs and sTJBEs during on-demand versus prophylaxis treatment were experienced by all 13 patients. Target joint sABR during prophylaxis was 0 for 5/13 patients. ABR reduction ranged from 66.1% (27.2→9.2) to 97.8% (46.2→1.0); sTJBE reductions ranged from 6.2% (2.1→2.0) to 100% (from 40.1, 19.1, 3.9, 9.0, 6.1--0). During prophylaxis, 47% (8/17) of trough FIX activity samples were more than 2%. In sBE patients, ABR and number of TJBEs were reduced with once-weekly nonacog alfa. When sBEs occurred, they followed no apparent pattern for day of occurrence. Clinicaltrials.gov identifier: NCT01335061.
(Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc.)

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