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

Transfusion

Volumn 55, Issue 7, 2015, Pages 1787-1797

  Polack, Benoît ^a,j   Calvez, Thierry ^b   Chambost, Hervé ^c   Rothschild, Chantal ^d   Goudemand, Jenny ^e   Claeyssens, Ségolène ^f   Borel Derlon, Annie ^g   Bardoulat, Isabelle ^h   Maurel, Frédérique ^h   Woronoff Lemsi, Marie Christine ⁱ  

^a GRENOBLE UNIVERSITY HOSPITAL   (France)

^b SORBONNE UNIVERSITÉ   (France)

^c AIX MARSEILLE UNIVERSITY   (France)

^d HÔPITAL NECKER ENFANTS MALADES   (France)

^e LILLE UNIVERSITY HOSPITAL   (France)

^f CHU PURPAN   (France)

^g CAEN UNIVERSITY HOSPITAL   (France)

^h IQVIA   (United States)

ⁱ UNIVERSITY HOSPITAL OF BESANÇON   (France)

^j UNIV GRENOBLE ALPES   (France)

more..

Author keywords

[No Author keywords available]

Indexed keywords

PLASMA DERIVED BLOOD CLOTTING FACTOR 9; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG; BLOOD CLOTTING FACTOR 9;

ADOLESCENT; ADULT; AGED; ARTICLE; BLEEDING; CHILD; COHORT ANALYSIS; DISEASE SEVERITY; DRUG COST; DRUG EFFICACY; DRUG USE; FEMALE; HEALTH CARE COST; HEALTH CARE PLANNING; HEMOPHILIA B; HUMAN; MAJOR CLINICAL STUDY; MALE; OBSERVATIONAL STUDY; QUALITY OF LIFE; CLINICAL TRIAL; COST; ECONOMICS; FRANCE; HEMORRHAGE; MIDDLE AGED; MULTICENTER STUDY;

ADOLESCENT; ADULT; CHILD; COHORT STUDIES; COSTS AND COST ANALYSIS; FACTOR IX; FEMALE; FRANCE; HEMOPHILIA B; HEMORRHAGE; HUMANS; MALE; MIDDLE AGED; QUALITY OF LIFE;

EID: 84937023941     PISSN: 00411132     EISSN: 15372995     Source Type: Journal    
DOI: 10.1111/trf.13016     Document Type: Article

References (28)

1. 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.
2. 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.
3. 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.
4. 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.
5. Escobar MA., Health economics in haemophilia: a review from the clinician's perspective. Haemophilia 2010; 16 Suppl 3: 29-34.
6. Feldman BM, Berger K, Bohn R, et al., Haemophilia prophylaxis: how can we justify the costs? Haemophilia 2012; 18: 680-4.
7. Mannucci PM, Tuddenham EG,. The hemophilias-from royal genes to gene therapy. N Engl J Med 2001; 344: 1773-9.
8. Berntorp E, Shapiro AD, Waters J, et al., The international factor IX treatment network survey. Haemophilia 2012; 18: e60 -2.
9. Santagostino E,. Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications. Haemophilia 2010; 16 Suppl 6: 13-7.
10. 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.
11. Gilbert MS., Prophylaxis: musculoskeletal evaluation. Semin Hematol 1993; 30 (3 Suppl 2): 3-6.
12. 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.
13. 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.
14. Guerois C, Lambert T, Peynet J, et al., Assessment of quality of life in hemophilia population: validation of the QUAL-HEMO, a French haemophilia age-group specific quality of life questionnaire. Haemophilia 2006; 12 (Suppl 2): PO804.
15. Agence Technique de l′Information sur l′Hospitalisation. Programme de médicalisation des systèmes d'information en médecine, chirurgie, obstétrique et odontologie (PMSI-MCO). Lyon: Agence Technique de l'Information sur l'Hospitalisation; 2013 [cited 2014 Jan 3]. Available from: http://www.atih.sante.fr/mco/presentation.
16. L′Assurance Maladie. Documentation technique. Paris: L'Assurance Maladie, Securite Sociale; [2011 Jan 1] Available from: http://www.ameli.fr/l-assurance-maladie/professionnels-de-sante.
17. 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.
18. Gater A, Thomson TA, Strandberg-Larsen M,. Haemophilia B: impact on patients and economic burden of disease. Thromb Haemost 2011; 106: 398-404.
19. Kisker CT, Eisberg A, Schwartz B,. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia 2003; 9: 279-84.
20. 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.
21. 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.
22. 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.
23. 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.
24. 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.
25. 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.
26. 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.
27. White GC, 2nd, Beebe A, Nielsen B,. Recombinant factor IX. Thromb Haemost 1997; 78: 261-5.
28. 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.