EQOFIX: a combined economic and quality-of-life study of hemophilia B treatments in France

BACKGROUND EQOFIX is a medicoeconomic study that analyzed the health‐related quality of life (HRQoL) and costs of care of the moderate and severe forms of hemophilia B, treated on demand or by prophylaxis with either plasma‐derived Factor IX (pdFIX) or recombinant FIX (rFIX). STUDY DESIGN AND METHOD...

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Published in:Transfusion Vol. 55; no. 7; pp. 1787 - 1797
Main Authors: Polack, Benoît, Calvez, Thierry, Chambost, Hervé, Rothschild, Chantal, Goudemand, Jenny, Claeyssens, Ségolène, Borel-Derlon, Annie, Bardoulat, Isabelle, Maurel, Frédérique, Woronoff-Lemsi, Marie-Christine
Format: Journal Article
Language:English
Published: United States Blackwell Publishing Ltd 01.07.2015
Wiley
Wiley Subscription Services, Inc
Subjects:
[INFO.INFO-BT]Computer Science [cs]/Biotechnology
Adolescent
Adult
Child
Cohort Studies
Costs
Costs and Cost Analysis
Economics and Finance
Factor IX
Factor IX - administration & dosage
Factor IX - economics
Female
France
Hematology
Hemophilia
Hemophilia B
Hemophilia B - drug therapy
Hemophilia B - economics
Hemorrhage
Hemorrhage - economics
Hemorrhage - prevention & control
Human health and pathology
Humanities and Social Sciences
Humans
Life Sciences
Male
Middle Aged
Quality of Life
Santé publique et épidémiologie
France
ISSN:0041-1132, 1537-2995, 1537-2995
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Summary:BACKGROUND EQOFIX is a medicoeconomic study that analyzed the health‐related quality of life (HRQoL) and costs of care of the moderate and severe forms of hemophilia B, treated on demand or by prophylaxis with either plasma‐derived Factor IX (pdFIX) or recombinant FIX (rFIX). STUDY DESIGN AND METHODS The primary objectives were evaluations of the impact of hemophilia B on HRQoL and of the costs associated with its management. The secondary objectives were evaluations of the clinical efficacy and costs of care of pdFIX and rFIX. In this observational study we included and followed for 1 year severe and moderate hemophilia B patients without inhibitor. HRQoL was evaluated through generic and disease‐specific questionnaires. Information on the health resources consumed was collected every 3 months. RESULTS The EQOFIX cohort was composed of 155 patients, including 51 children and 104 adults, with 114 having severe disease and 41 having moderate disease. The regimens were prophylactic for 61 and on demand for 94. Altogether, 78 were treated with rFIX and 77 with pdFIX. There was no difference in the QoL between the pdFIX and rFIX treatments. The extra cost of prophylaxis was €22,605 per bleeding event prevented. The consumption of FIX was 1.4‐fold higher for the patients treated with rFIX than for the patients treated with pdFIX. CONCLUSION Our findings in a cohort composed of 25% of the French population of moderate and severe hemophilia B patients show, with similar clinical and HRQoL results, that treatment with rFIX is more expensive than treatment with pdFIX.
Bibliography:Supported by Laboratoire Français du Fractionnement et des Biotechnologies (LFB). The funding source had no role in the study design; in the collection, analysis or interpretation of the data; in the writing of the report; or in the decision to submit this paper
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ArticleID:TRF13016
ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
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ISSN:0041-1132
1537-2995
1537-2995
DOI:10.1111/trf.13016