| Reformulated BeneFix: efficacy and safety in previously treated patients with moderately severe to severe haemophilia B. | |
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MedLine Citation:
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PMID: 17498071 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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BeneFix, the only recombinant factor IX (FIX), has been reformulated. The reformulation involves a change in diluent and allows for more concentrated infusions of recombinant FIX. A double-blind, randomized, pharmacokinetic (PK) crossover study demonstrated that reformulated BeneFix was bioequivalent to original BeneFix and follow-up PK evaluation after 6 months of treatment demonstrated the PK stability of reformulated BeneFix after multiple exposures. Favourable efficacy and safety profiles, consistent with those already well-established for original BeneFix, were observed: 81.1% of haemorrhages resolved with only a single infusion; 85.3% of initial treatment response ratings were Excellent or Good; more than half of the subjects using reformulated BeneFix for routine prophylaxis (11 of 17, 64.7%) had no spontaneous haemorrhages during their 6-12 month course of prophylactic treatment, with an overall spontaneous bleeding rate of 0.72 year(-1); and for the single surgical procedure (knee washing), treatment was rated Useful. In addition, there was no FIX inhibitor development, allergic-type manifestations, or thrombogenic complications with more than 1100 infusions (nearly 5.2 million IUs) administered in this trial. All efficacy and safety outcomes from this study were achieved with more concentrated recombinant protein infusions than that possible with original BeneFix, and utilization of the 2000 IU per vial dosage strength, newly introduced with the reformulated product, was high (>62%). The reformulation of BeneFix allows smaller delivery volumes and an increased choice of dosage strengths without altering the PK properties (including incremental recovery and half-life) or the established efficacy and safety profile of recombinant FIX. |
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Authors:
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T Lambert; M Recht; L A Valentino; J S Powell; C Udata; S T Sullivan; D A Roth |
Publication Detail:
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Type: Journal Article; Research Support, Non-U.S. Gov't |
Journal Detail:
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Title: Haemophilia : the official journal of the World Federation of Hemophilia Volume: 13 ISSN: 1351-8216 ISO Abbreviation: Haemophilia Publication Date: 2007 May |
Date Detail:
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Created Date: 2007-05-14 Completed Date: 2007-09-26 Revised Date: 2009-10-21 |
Medline Journal Info:
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Nlm Unique ID: 9442916 Medline TA: Haemophilia Country: England |
Other Details:
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Languages: eng Pagination: 233-43 Citation Subset: IM |
Affiliation:
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Centre de Traitement des Hémophiles, Hôpital de Bicêtre AP-HP, Le Kremlin Bicêtre, France. thierry.lambert@bct.aphp.fr |
Export Citation:
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APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
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Adolescent Adult Child Cross-Over Studies Double-Blind Method Factor IX / therapeutic use* Hemarthrosis / prevention & control* Hemophilia B / drug therapy* Hemorrhage / prevention & control* Humans Middle Aged Randomized Controlled Trials as Topic Recombinant Proteins / therapeutic use* |
| Chemical | |
Reg. No./Substance:
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0/Recombinant Proteins; 9001-28-9/Factor IX |
| Comments/Corrections | |
Erratum In:
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Haemophilia. 2007 Jul;13(4):450 |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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