Systematic review and analysis of efficacy of recombinant factor IX products for prophylactic treatment of hemophilia B in comparison with rIX-FP.

Détails

ID Serval
serval:BIB_4D4C4D16EF89
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
Systematic review and analysis of efficacy of recombinant factor IX products for prophylactic treatment of hemophilia B in comparison with rIX-FP.
Périodique
Journal of medical economics
Auteur⸱e⸱s
Davis J., Yan S., Matsushita T., Alberio L., Bassett P., Santagostino E.
ISSN
1941-837X (Electronic)
ISSN-L
1369-6998
Statut éditorial
Publié
Date de publication
10/2019
Peer-reviewed
Oui
Volume
22
Numéro
10
Pages
1014-1021
Langue
anglais
Notes
Publication types: Journal Article ; Systematic Review
Publication Status: ppublish
Résumé
Aims: Prophylaxis with standard-acting recombinant factor IX (rFIX) in hemophilia B patients requires frequent injections. Extended half-life (EHL) products allow for prolonged dosing intervals, and so reduce this treatment burden. Three technologies are employed to extend the half-life of FIX; glycopegylation, Fc-fusion, and albumin fusion. rIX-FP is a novel albumin fusion protein, which allows for a prolonged dosing interval of up to 14 days. A systematic review and indirect statistical comparison was performed to evaluate the efficacy of both EHL and standard-acting rFIX products compared with rIX-FP in Phase III trials for prophylaxis in adult hemophilia B patients. Materials and methods: A systematic search was conducted in both EMBASE and PubMed to identify Phase III trials of prophylactic rFIX treatment in previously treated hemophilia B patients aged ≥12 years (FIX ≤2%). Annualized bleeding rate (ABR), spontaneous ABR (AsBR), and joint ABR (AjBR) data were extracted from each study. A z-test was performed using the mean of each parameter, and the mean difference in outcome between studies was calculated. Results: Seven articles investigating six rFIX products were identified. Median ABR, AsBR, and AjBR ranged from 0-3.0, 0-1.0, and 0-1.1 (means = 0.8-4.26, 0.13-2.6, and 0.34-2.85), respectively. rIX-FP achieved the lowest median and mean values in all three parameters. Z-tests showed that mean ABR was significantly lower for rIX-FP 7-day prophylaxis compared with the majority of standard-acting and other EHL rFIX products. Limitations: The low number of appropriate trials available for comparison limits the quantity of data available for comparison, and restricts the use of methods of adjustment for variance in study design or patient characteristics. However, these limitations are shared with similar analyses published in this field. Conclusion: This indirect comparison of Phase III trials indicates that rIX-FP efficacy compares favorably vs other rFIX products for prophylaxis in hemophilia B.
Mots-clé
Clinical Trials, Phase III as Topic, Factor IX/administration & dosage, Hemophilia B/drug therapy, Humans, Recombinant Fusion Proteins/administration & dosage, Treatment Outcome, Factor IX, I12, I19, annualized bleed rate, hemophilia B, prophylaxis, recombinant, systematic review
Pubmed
Web of science
Open Access
Oui
Création de la notice
25/05/2019 13:18
Dernière modification de la notice
28/02/2020 6:19
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