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

Benoît Polack (), Thierry Calvez, Hervé Chambost, Chantal Rothschild, Jenny Goudemand, Ségolène Claeyssens, Annie Borel-Derlon, Isabelle Bardoulat, Frédérique Maurel and Marie-Christine Woronoff-Lemsi
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Benoît Polack: TIMC-IMAG - Techniques de l'Ingénierie Médicale et de la Complexité - Informatique, Mathématiques et Applications, Grenoble - UMR 5525 - UJF - Université Joseph Fourier - Grenoble 1 - Grenoble INP - Institut polytechnique de Grenoble - Grenoble Institute of Technology - VAS - VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement - CNRS - Centre National de la Recherche Scientifique
Thierry Calvez: UPMC - Université Pierre et Marie Curie - Paris 6 - UFR de Médecine Pierre et Marie Curie - UPMC - Université Pierre et Marie Curie - Paris 6, iPLESP - Institut Pierre Louis d'Epidémiologie et de Santé Publique - UPMC - Université Pierre et Marie Curie - Paris 6 - INSERM - Institut National de la Santé et de la Recherche Médicale
Hervé Chambost: Service de pédiatrie spécialisée et médecine infantile (neurologie, pneumologie, maladies héréditaires du métabolisme) [Hôpital de la Timone - APHM] - TIMONE - Hôpital de la Timone [CHU - APHM]
Chantal Rothschild: Service d'immuno-hématologie pédiatrique [CHU Necker] - AP-HP - Assistance publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker - Enfants Malades [AP-HP] - AP-HP - Assistance publique - Hôpitaux de Paris (AP-HP)
Jenny Goudemand: Hôpital cardiologique - Université de Lille, Droit et Santé - CHRU Lille - Centre Hospitalier Régional Universitaire [CHU Lille]
Ségolène Claeyssens: ADEN - Nutrition, inflammation et dysfonctionnement de l'axe intestin-cerveau - UNIROUEN - Université de Rouen Normandie - NU - Normandie Université - INSERM - Institut National de la Santé et de la Recherche Médicale
Annie Borel-Derlon: Sérine protéases et physiopathologie de l'unité neurovasculaire - UNICAEN - Université de Caen Normandie - NU - Normandie Université - INSERM - Institut National de la Santé et de la Recherche Médicale
Marie-Christine Woronoff-Lemsi: RIGHT - Interactions hôte-greffon-tumeur, ingénierie cellulaire et génique - UFC (UMR INSERM 1098) - INSERM - Institut National de la Santé et de la Recherche Médicale - EFS BFC - Etablissement français du sang [Bourgogne-Franche-Comté] - UFC - Université de Franche-Comté - UBFC - Université Bourgogne Franche-Comté [COMUE]

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Abstract: 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.

Date: 2015
Note: View the original document on HAL open archive server: https://hal.science/hal-01463671v1
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Published in Transfusion, 2015, 55 (7), pp.1787-1797. ⟨10.1111/trf.13016⟩

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Persistent link: https://EconPapers.repec.org/RePEc:hal:journl:hal-01463671

DOI: 10.1111/trf.13016

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