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 |
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| Main Authors: | , , , , , , , , , |
| Format: | Journal Article |
| Language: | English |
| Published: |
United States
Blackwell Publishing Ltd
01.07.2015
Wiley Wiley Subscription Services, Inc |
| Subjects: | |
| ISSN: | 0041-1132, 1537-2995, 1537-2995 |
| Online Access: | Get full text |
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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. |
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| AbstractList | 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 [euro]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. 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. 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. 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). 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. 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. 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. 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).BACKGROUNDEQOFIX 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).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.STUDY DESIGN AND METHODSThe 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.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.RESULTSThe 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.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.CONCLUSIONOur 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. |
| Author | Maurel, Frédérique Bardoulat, Isabelle Woronoff-Lemsi, Marie-Christine Calvez, Thierry Chambost, Hervé Goudemand, Jenny Borel-Derlon, Annie Rothschild, Chantal Polack, Benoît Claeyssens, Ségolène |
| Author_xml | – sequence: 1 givenname: Benoît surname: Polack fullname: Polack, Benoît email: BPolack@chu-grenoble.fr organization: Department of Hematology, University Hospital, and CNRS UMR5525, Grenoble-Alpes University, Grenoble, France – sequence: 2 givenname: Thierry surname: Calvez fullname: Calvez, Thierry organization: Sorbonne University, UPMC University of Paris 06, INSERM, UMR-S 1136, Pierre Louis Institute of Epidemiology and Public Health, Paris, France – sequence: 3 givenname: Hervé surname: Chambost fullname: Chambost, Hervé organization: APHM, Department of Pediatric Hematology, Children Hospital La Timone, and INSERM, UMR 1062, Aix-Marseille University, Marseille, France – sequence: 4 givenname: Chantal surname: Rothschild fullname: Rothschild, Chantal organization: Regional Hemophilia Center, Department of Hematology, University Hospital Necker Enfants Malades, Paris, France – sequence: 5 givenname: Jenny surname: Goudemand fullname: Goudemand, Jenny organization: Department of Hematology-Transfusion, University Hospital, Lille, France – sequence: 6 givenname: Ségolène surname: Claeyssens fullname: Claeyssens, Ségolène organization: Regional Hemophilia Center, Purpan Hospital, Toulouse, France – sequence: 7 givenname: Annie surname: Borel-Derlon fullname: Borel-Derlon, Annie organization: Regional Hemophilia and VWD Center, University Hospital, and INSERM U919, Caen, France – sequence: 8 givenname: Isabelle surname: Bardoulat fullname: Bardoulat, Isabelle organization: Department of Health Economics and Outcome Research, IMS Health, La Défense, France – sequence: 9 givenname: Frédérique surname: Maurel fullname: Maurel, Frédérique organization: Department of Health Economics and Outcome Research, IMS Health, La Défense, France – sequence: 10 givenname: Marie-Christine surname: Woronoff-Lemsi fullname: Woronoff-Lemsi, Marie-Christine organization: Pharmacy, University Hospital, and INSERM UMR1098, Franche-Comté University, Besançon, France |
| BackLink | https://cir.nii.ac.jp/crid/1873961342779374464$$DView record in CiNii https://www.ncbi.nlm.nih.gov/pubmed/25652955$$D View this record in MEDLINE/PubMed https://hal.science/hal-01463671$$DView record in HAL |
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| CODEN | TRANAT |
| CitedBy_id | crossref_primary_10_1111_trf_13988 crossref_primary_10_1111_hae_70101 crossref_primary_10_1016_j_omtm_2019_09_005 crossref_primary_10_1182_blood_2017_09_804419 crossref_primary_10_1016_j_pharma_2022_11_001 crossref_primary_10_1016_j_jval_2017_04_017 crossref_primary_10_1111_hae_14755 crossref_primary_10_1111_hae_14941 crossref_primary_10_1111_hae_14129 crossref_primary_10_1016_j_jtha_2024_05_026 crossref_primary_10_1186_s12913_022_07626_x crossref_primary_10_1007_s40273_017_0588_z crossref_primary_10_1080_14737167_2025_2532805 crossref_primary_10_1111_hae_14620 |
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| ContentType | Journal Article |
| Contributor | Sérine protéases et physiopathologie de l'unité neurovasculaire Sérine protéases et physiopathologie de l'unité neurovasculaire ; Université de Caen Normandie (UNICAEN) ; Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM) Techniques de l'Ingénierie Médicale et de la Complexité - Informatique, Mathématiques et Applications, Grenoble - UMR 5525 (TIMC-IMAG) ; VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-Centre National de la Recherche Scientifique (CNRS)-Université Joseph Fourier - Grenoble 1 (UJF) Interactions hôte-greffon-tumeur, ingénierie cellulaire et génique - UFC (UMR INSERM 1098) (RIGHT) Service de pédiatrie spécialisée et médecine infantile (neurologie, pneumologie, maladies héréditaires du métabolisme) Hôpital de la Timone Schved, Jean-François Rafowicz, Anne Pignon, Bernard Stieltjes, Nathalie Wibaut, Bénédicte Rothschild, Chantal d'Oiron, Roseline Gembara, Piort Peynet, Jocelyne Charbonneau, Sylvie Hassoun, Abel Faradji, Albert Bicetre, Le Kremlin Soler, Catherine Borel-Derlon, Annie Goudemand, Jenny Fimbel, Béatrice Ancelet, Daria Gorde, Stéphanie Aouba, Achile Volot, Fabienne Gruel, Yves Trossaert, Marc Torchet, Marie-Françoise Berger, Claire Guerin, Viviane Navaro, Robert Ounnoughène, Nadra Bettembourg, Armelle Polack, Benoît Milien, Vanessa Meunier, Sandrine Guillet, Benoît Lambert, Thierry Marquès-Verdier, Alain Briquel, Marie-Elisabeth Durin-Assolant, Anne Hézard, Nathalie Claeyssens, Ségolène Biron- Andréani, Christine Donadio, Daniel Ternisien, Catherine Borg, Jeanne-Yvonne Bertrand, Marie-Anne Pan-Petesch, Brigitte Le Cam Duchez, Véronique Matingou, Marceline Albinni, Souha Lutz, Patrick Bouvet, Nadine Sigaud, Marianne Nguyen, Philippe Fribault, Anne Gay, Valérie Guillon, Patricia Roussel-Robert, Valérie Négrier, Claude Chambost, Hervé Chamouni, Pier |
| Contributor_xml | – sequence: 1 fullname: Techniques de l'Ingénierie Médicale et de la Complexité - Informatique, Mathématiques et Applications, Grenoble - UMR 5525 (TIMC-IMAG) ; Université Joseph Fourier - Grenoble 1 (UJF)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Centre National de la Recherche Scientifique (CNRS) – sequence: 1 givenname: Marie-Anne surname: Bertrand fullname: Bertrand, Marie-Anne – sequence: 2 fullname: Laboratoire d'hématologie – sequence: 2 givenname: Viviane surname: Guerin fullname: Guerin, Viviane – sequence: 3 fullname: Université Pierre et Marie Curie - Paris 6 - UFR de Médecine Pierre et Marie Curie (UPMC) ; Université Pierre et Marie Curie - Paris 6 (UPMC) – sequence: 3 givenname: Brigitte surname: Pan-Petesch fullname: Pan-Petesch, Brigitte – sequence: 4 fullname: Institut Pierre Louis d'Epidémiologie et de Santé Publique (iPLESP) ; Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM) – sequence: 4 givenname: Annie surname: Borel-Derlon fullname: Borel-Derlon, Annie – sequence: 5 fullname: Service de pédiatrie spécialisée et médecine infantile (neurologie, pneumologie, maladies héréditaires du métabolisme) [Hôpital de la Timone - APHM] ; Hôpital de la Timone [CHU - APHM] (TIMONE) – sequence: 5 givenname: Patricia surname: Guillon fullname: Guillon, Patricia – sequence: 6 fullname: Service d'immuno-hématologie pédiatrique [CHU Necker] ; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Necker - Enfants Malades [AP-HP] ; Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP) – sequence: 6 givenname: Valérie surname: Gay fullname: Gay, Valérie – sequence: 7 fullname: Hôpital cardiologique ; Université de Lille, Droit et Santé-Centre Hospitalier Régional Universitaire [CHU Lille] (CHRU Lille) – sequence: 7 givenname: Alain surname: Marquès-Verdier fullname: Marquès-Verdier, Alain – sequence: 8 fullname: Nutrition, inflammation et dysfonctionnement de l'axe intestin-cerveau (ADEN) ; Université de Rouen Normandie (UNIROUEN) ; Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM) – sequence: 8 givenname: Piort surname: Gembara fullname: Gembara, Piort – sequence: 9 fullname: Sérine protéases et physiopathologie de l'unité neurovasculaire ; Université de Caen Normandie (UNICAEN) ; Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM) – sequence: 9 givenname: Fabienne surname: Volot fullname: Volot, Fabienne – sequence: 10 fullname: Department of Health Economics and Outcome Research, IMS Health, La Défense, France – sequence: 10 givenname: Benoît surname: Polack fullname: Polack, Benoît – sequence: 11 fullname: Interactions hôte-greffon-tumeur, ingénierie cellulaire et génique - UFC (UMR INSERM 1098) (RIGHT) ; Institut National de la Santé et de la Recherche Médicale (INSERM)-Etablissement français du sang [Bourgogne-Franche-Comté] (EFS BFC)-Université de Franche-Comté (UFC) ; Université Bourgogne Franche-Comté [COMUE] (UBFC)-Université Bourgogne Franche-Comté [COMUE] (UBFC) – sequence: 11 givenname: Jocelyne surname: Peynet fullname: Peynet, Jocelyne – sequence: 12 fullname: Techniques de l'Ingénierie Médicale et de la Complexité - Informatique, Mathématiques et Applications, Grenoble - UMR 5525 (TIMC-IMAG) ; VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-Centre National de la Recherche Scientifique (CNRS)-Université Joseph Fourier - Grenoble 1 (UJF) – sequence: 12 givenname: Le Kremlin surname: Bicetre fullname: Bicetre, Le Kremlin – sequence: 13 fullname: Service d'Hématologie pédiatrique, Hôpital de la Timone, Marseille ; Assistance Publique - 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Informatique, Mathématiques et Applications, Grenoble - UMR 5525 (TIMC-IMAG) – sequence: 18 givenname: Nathalie surname: Trillot fullname: Trillot, Nathalie – sequence: 19 fullname: Université Joseph Fourier - Grenoble 1 (UJF)-Institut polytechnique de Grenoble - Grenoble Institute of Technology (Grenoble INP )-VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Centre National de la Recherche Scientifique (CNRS) – sequence: 19 givenname: Bénédicte surname: Wibaut fullname: Wibaut, Bénédicte – sequence: 20 fullname: Université Pierre et Marie Curie - Paris 6 - UFR de Médecine Pierre et Marie Curie (UPMC) – sequence: 20 givenname: Caroline surname: Oudot fullname: Oudot, Caroline – sequence: 21 fullname: Université Pierre et Marie Curie - Paris 6 (UPMC) – sequence: 21 givenname: Claude surname: Négrier fullname: Négrier, Claude – sequence: 22 fullname: Institut Pierre Louis d'Epidémiologie et de Santé Publique (iPLESP) – sequence: 22 givenname: Anne surname: Durin-Assolant fullname: Durin-Assolant, Anne – sequence: 23 fullname: Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM) – sequence: 23 givenname: Anne-Marie surname: Lienhart fullname: Lienhart, Anne-Marie – sequence: 24 fullname: Service de pédiatrie spécialisée et médecine infantile (neurologie, pneumologie, maladies héréditaires du métabolisme) Hôpital de la Timone - APHM – sequence: 24 givenname: Sandrine surname: Meunier fullname: Meunier, Sandrine – sequence: 25 fullname: Hôpital de la Timone CHU - APHM (TIMONE) – sequence: 25 givenname: Hervé surname: Chambost fullname: Chambost, Hervé – sequence: 26 fullname: Service d'immuno-hématologie pédiatrique CHU Necker – sequence: 26 givenname: Vanessa surname: Milien fullname: Milien, Vanessa – sequence: 27 fullname: Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Necker - Enfants Malades AP-HP – sequence: 27 givenname: Abel surname: Hassoun fullname: Hassoun, Abel – sequence: 28 fullname: Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP) – sequence: 28 givenname: Jean-François surname: Schved fullname: Schved, Jean-François – sequence: 29 fullname: Hôpital cardiologique – sequence: 29 givenname: Christine surname: Biron- Andréani fullname: Biron- Andréani, Christine – sequence: 30 fullname: Université de Lille, Droit et Santé-Centre Hospitalier Régional Universitaire Lille (CHRU Lille) – sequence: 30 givenname: Daniel surname: Donadio fullname: Donadio, Daniel – sequence: 31 fullname: Nutrition, inflammation et dysfonctionnement de l'axe intestin-cerveau (ADEN) – sequence: 31 givenname: Robert surname: Navaro fullname: Navaro, Robert – sequence: 32 fullname: Université de Rouen Normandie (UNIROUEN) – sequence: 32 givenname: Marie-Elisabeth surname: Briquel fullname: Briquel, Marie-Elisabeth – sequence: 33 fullname: Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM) – sequence: 33 givenname: Marc surname: Trossaert fullname: Trossaert, Marc – sequence: 34 fullname: Sérine protéases et physiopathologie de l'unité neurovasculaire – sequence: 34 givenname: Daria surname: Ancelet fullname: Ancelet, Daria – sequence: 35 fullname: Université de Caen Normandie (UNICAEN) – sequence: 35 givenname: Marianne surname: Sigaud fullname: Sigaud, Marianne – sequence: 36 fullname: Interactions hôte-greffon-tumeur, ingénierie cellulaire et génique - 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| Copyright | 2015 The Authors. Transfusion published by Wiley Periodicals, Inc. on behalf of AABB. 2015 AABB Distributed under a Creative Commons Attribution 4.0 International License |
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| References | Berntorp E, Shapiro AD, Waters J, et al. The international factor IX treatment network survey. Haemophilia 2012;18:e60-2. Gringeri A, Mantovani LG, Scalone L, et al. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003;102:2358-63. Neumann PJ, Cohen JT, Weinstein MC. Updating cost-effectiveness-the curious resilience of the $50,000-per-QALY threshold. N Engl J Med 2014;371:796-7. Barr RD, Saleh M, Furlong W, et al. Health status and health-related quality of life associated with hemophilia. Am J Hematol 2002;71:152-60. Herdman M, Rajmil L, Ravens-Sieberer U, et al. Expert consensus in the development of a European health-related quality of life measure for children and adolescents: a Delphi study. Acta Paediatr 2002;91:1385-90. Miners AH, Sabin CA, Stevens AJ, et al. Financing the rising cost of haemophilia care at a large comprehensive care centre. J R Coll Phys Lond 1997;31:640-4. Kisker CT, Eisberg A, Schwartz B. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia 2003;9:279-84. Leplège A, Ecosse E, Pouchot J, et al. Le questionnaire SF-36. Manuel de l'utilisateur et guide d'interprétation des scores. Paris: ESTEM; 2001. Gilbert MS. Prophylaxis: musculoskeletal evaluation. Semin Hematol 1993;30(3 Suppl 2):3-6. Escobar MA. Health economics in haemophilia: a review from the clinician's perspective. Haemophilia 2010;16 Suppl 3:29-34. Gater A, Thomson TA, Strandberg-Larsen M. Haemophilia B: impact on patients and economic burden of disease. Thromb Haemost 2011;106:398-404. White GC 2nd, Beebe A, Nielsen B. Recombinant factor IX. Thromb Haemost 1997;78:261-5. Brorson K, De Wit C, Hamilton E, et al. Impact of cell culture process changes on endogenous retrovirus expression. Biotechnol Bioeng 2002;80:257-67. Ware JE Jr, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care 1992;30:473-83. Nicholson A, Berger K, Bohn R, et al. Recommendations for reporting economic evaluations of haemophilia prophylaxis: a nominal groups consensus statement on behalf of the Economics Expert Working Group of The International Prophylaxis Study Group. Haemophilia 2008;14:127-32. Santagostino E. Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications. Haemophilia 2010;16 Suppl 6:13-7. Tencer T, Friedman HS, Li-McLeod J, et al. Medical costs and resource utilization for hemophilia patients with and without HIV or HCV infection. J Manag Care Pharm 2007;13:790-8. Szucs TD, Offner A, Kroner B, et al. Resource utilisation in haemophiliacs treated in Europe: results from the European Study on Socioeconomic Aspects of Haemophilia Care. The European Socioeconomic Study Group. Haemophilia 1998;4:498-501. den Uijl IE, Fischer K, Van Der Bom JG, et al. Clinical outcome of moderate haemophilia compared with severe and mild haemophilia. Haemophilia 2009;15:83-90. Stieltjes N, Torchet MF, Misrahi L, et al. Epidemiological survey of haemophiliacs with inhibitors in France: orthopaedic status, quality of life and cost-the 'Statut Orthopedique des Patients Hemophiles' avec Inhibiteur study. Blood Coagul Fibrinolysis 2009;20:4-11. Mannucci PM, Tuddenham EG. The hemophilias-from royal genes to gene therapy. N Engl J Med 2001;344:1773-9. Aledort L, Bullinger M, von Mackensen S, et al. Why should we care about quality of life in persons with haemophilia? Haemophilia 2012;18:e154-7. Feldman BM, Berger K, Bohn R, et al. Haemophilia prophylaxis: how can we justify the costs? Haemophilia 2012;18:680-4. Ewenstein BM, Joist JH, Shapiro AD, et al. Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B. Transfusion 2002;42:190-7. Germain L, Erpelding M, Doncarli A, et al. Health related quality of life in French haemophilia population: a comparative analysis with French general population. Haemophilia 2010;16(Suppl 4):136-7. 2001; 344 2010; 16 2011; 106 2009; 20 2006; 12 2001 2002; 42 1997; 31 1993; 30 2003; 9 1997; 78 2008; 14 2012; 18 2002; 71 2014; 371 2002; 91 2002; 80 2003; 102 1998; 4 2007; 13 2009; 15 1992; 30 e_1_2_8_28_1 e_1_2_8_29_1 e_1_2_8_24_1 e_1_2_8_25_1 e_1_2_8_26_1 e_1_2_8_27_1 e_1_2_8_3_1 e_1_2_8_2_1 e_1_2_8_4_1 e_1_2_8_7_1 e_1_2_8_6_1 e_1_2_8_9_1 e_1_2_8_8_1 e_1_2_8_20_1 e_1_2_8_22_1 e_1_2_8_23_1 e_1_2_8_17_1 e_1_2_8_19_1 Miners AH (e_1_2_8_5_1) 1997; 31 e_1_2_8_13_1 e_1_2_8_14_1 e_1_2_8_15_1 e_1_2_8_16_1 Leplège A (e_1_2_8_18_1) 2001 Germain L (e_1_2_8_21_1) 2010; 16 e_1_2_8_10_1 e_1_2_8_11_1 Gilbert MS. (e_1_2_8_12_1) 1993; 30 |
| References_xml | – reference: Santagostino E. Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications. Haemophilia 2010;16 Suppl 6:13-7. – reference: Herdman M, Rajmil L, Ravens-Sieberer U, et al. Expert consensus in the development of a European health-related quality of life measure for children and adolescents: a Delphi study. Acta Paediatr 2002;91:1385-90. – reference: Aledort L, Bullinger M, von Mackensen S, et al. Why should we care about quality of life in persons with haemophilia? Haemophilia 2012;18:e154-7. – reference: Gater A, Thomson TA, Strandberg-Larsen M. Haemophilia B: impact on patients and economic burden of disease. Thromb Haemost 2011;106:398-404. – reference: Barr RD, Saleh M, Furlong W, et al. Health status and health-related quality of life associated with hemophilia. Am J Hematol 2002;71:152-60. – reference: Szucs TD, Offner A, Kroner B, et al. Resource utilisation in haemophiliacs treated in Europe: results from the European Study on Socioeconomic Aspects of Haemophilia Care. The European Socioeconomic Study Group. Haemophilia 1998;4:498-501. – reference: Gringeri A, Mantovani LG, Scalone L, et al. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003;102:2358-63. – reference: Nicholson A, Berger K, Bohn R, et al. Recommendations for reporting economic evaluations of haemophilia prophylaxis: a nominal groups consensus statement on behalf of the Economics Expert Working Group of The International Prophylaxis Study Group. Haemophilia 2008;14:127-32. – reference: Berntorp E, Shapiro AD, Waters J, et al. The international factor IX treatment network survey. Haemophilia 2012;18:e60-2. – reference: Leplège A, Ecosse E, Pouchot J, et al. Le questionnaire SF-36. Manuel de l'utilisateur et guide d'interprétation des scores. Paris: ESTEM; 2001. – reference: Escobar MA. Health economics in haemophilia: a review from the clinician's perspective. Haemophilia 2010;16 Suppl 3:29-34. – reference: White GC 2nd, Beebe A, Nielsen B. Recombinant factor IX. Thromb Haemost 1997;78:261-5. – reference: Miners AH, Sabin CA, Stevens AJ, et al. Financing the rising cost of haemophilia care at a large comprehensive care centre. J R Coll Phys Lond 1997;31:640-4. – reference: Kisker CT, Eisberg A, Schwartz B. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia 2003;9:279-84. – reference: Brorson K, De Wit C, Hamilton E, et al. Impact of cell culture process changes on endogenous retrovirus expression. Biotechnol Bioeng 2002;80:257-67. – reference: den Uijl IE, Fischer K, Van Der Bom JG, et al. Clinical outcome of moderate haemophilia compared with severe and mild haemophilia. Haemophilia 2009;15:83-90. – reference: Neumann PJ, Cohen JT, Weinstein MC. Updating cost-effectiveness-the curious resilience of the $50,000-per-QALY threshold. N Engl J Med 2014;371:796-7. – reference: Mannucci PM, Tuddenham EG. The hemophilias-from royal genes to gene therapy. N Engl J Med 2001;344:1773-9. – reference: Stieltjes N, Torchet MF, Misrahi L, et al. Epidemiological survey of haemophiliacs with inhibitors in France: orthopaedic status, quality of life and cost-the 'Statut Orthopedique des Patients Hemophiles' avec Inhibiteur study. Blood Coagul Fibrinolysis 2009;20:4-11. – reference: Tencer T, Friedman HS, Li-McLeod J, et al. Medical costs and resource utilization for hemophilia patients with and without HIV or HCV infection. J Manag Care Pharm 2007;13:790-8. – reference: Ware JE Jr, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care 1992;30:473-83. – reference: Germain L, Erpelding M, Doncarli A, et al. Health related quality of life in French haemophilia population: a comparative analysis with French general population. Haemophilia 2010;16(Suppl 4):136-7. – reference: Feldman BM, Berger K, Bohn R, et al. Haemophilia prophylaxis: how can we justify the costs? Haemophilia 2012;18:680-4. – reference: Gilbert MS. Prophylaxis: musculoskeletal evaluation. Semin Hematol 1993;30(3 Suppl 2):3-6. – reference: Ewenstein BM, Joist JH, Shapiro AD, et al. Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B. Transfusion 2002;42:190-7. – volume: 106 start-page: 398 year: 2011 end-page: 404 article-title: Haemophilia B: impact on patients and economic burden of disease publication-title: Thromb Haemost – volume: 102 start-page: 2358 year: 2003 end-page: 63 article-title: Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group publication-title: Blood – volume: 12 start-page: PO804 issue: Suppl 2 year: 2006 article-title: Assessment of quality of life in hemophilia population: validation of the QUAL‐HEMO, a French haemophilia age‐group specific quality of life questionnaire – volume: 91 start-page: 1385 year: 2002 end-page: 90 article-title: Expert consensus in the development of a European health‐related quality of life measure for children and adolescents: a Delphi study publication-title: Acta Paediatr – volume: 16 start-page: 13 issue: Suppl 6 year: 2010 end-page: 7 article-title: Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications publication-title: Haemophilia – volume: 18 start-page: e154 year: 2012 end-page: 7 article-title: Why should we care about quality of life in persons with haemophilia? publication-title: Haemophilia – volume: 4 start-page: 498 year: 1998 end-page: 501 article-title: Resource utilisation in haemophiliacs treated in Europe: results from the European Study on Socioeconomic Aspects of Haemophilia Care. The European Socioeconomic Study Group publication-title: Haemophilia – volume: 9 start-page: 279 year: 2003 end-page: 84 article-title: Prophylaxis in factor IX deficiency product and patient variation publication-title: Haemophilia – year: 2001 – volume: 18 start-page: 680 year: 2012 end-page: 4 article-title: Haemophilia prophylaxis: how can we justify the costs? publication-title: Haemophilia – volume: 344 start-page: 1773 year: 2001 end-page: 9 article-title: The hemophilias—from royal genes to gene therapy publication-title: N Engl J Med – volume: 16 start-page: 136 issue: Suppl 4 year: 2010 end-page: ‐7 article-title: Health related quality of life in French haemophilia population: a comparative analysis with French general population publication-title: Haemophilia – volume: 14 start-page: 127 year: 2008 end-page: 32 article-title: Recommendations for reporting economic evaluations of haemophilia prophylaxis: a nominal groups consensus statement on behalf of the Economics Expert Working Group of The International Prophylaxis Study Group publication-title: Haemophilia – volume: 371 start-page: 796 year: 2014 end-page: 7 article-title: Updating cost‐effectiveness—the curious resilience of the $50,000‐per‐QALY threshold publication-title: N Engl J Med – volume: 15 start-page: 83 year: 2009 end-page: 90 article-title: Clinical outcome of moderate haemophilia compared with severe and mild haemophilia publication-title: Haemophilia – volume: 80 start-page: 257 year: 2002 end-page: 67 article-title: Impact of cell culture process changes on endogenous retrovirus expression publication-title: Biotechnol Bioeng – volume: 30 start-page: 473 year: 1992 end-page: 83 article-title: The MOS 36‐item short‐form health survey (SF‐36). I. 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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... 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,... 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... 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... |
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| SubjectTerms | [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 |
| Title | EQOFIX: a combined economic and quality-of-life study of hemophilia B treatments in France |
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