The socioeconomic burden of patients affected by hemophilia with inhibitors

被引:47
作者
D'Angiolella, Lucia S. [1 ]
Cortesi, Paolo A. [1 ]
Rocino, Angiola [2 ]
Coppola, Antonio [3 ]
Hassan, Hamisa J. [4 ]
Giampaolo, Adele [4 ]
Solimeno, Luigi P. [5 ]
Lafranconi, Alessandra [1 ]
Micale, Mariangela [1 ]
Mangano, Sveva [1 ]
Crotti, Giacomo [1 ]
Pagliarin, Federica [1 ]
Cesana, Giancarlo [1 ]
Mantovani, Lorenzo G. [1 ]
机构
[1] Univ Milano Bicocca, Res Ctr Publ Hlth CESP, Monza, Italy
[2] San Giovanni Bosco Hosp, Hemophilia & Thrombosis Ctr, Naples, Italy
[3] Univ Hosp Parma, Reg Reference Ctr Inherited Bleeding Disorders, Parma, Italy
[4] Ist Super Sanita, Dept Oncol & Mol Med, Rome, Italy
[5] Fdn IRCCS Ca Granda Osped Maggiore Policlin, Div Orthopaed Surg & Traumatol, Milan, Italy
关键词
cost; hemophilia; inhibitors; quality of life; QUALITY-OF-LIFE; ACTIVATED FACTOR-VII; IMMUNE TOLERANCE INDUCTION; RECOMBINANT FACTOR-VIIA; PROTHROMBIN COMPLEX CONCENTRATE; MODERATE BLEEDING EPISODES; ELECTIVE ORTHOPEDIC-SURGERY; PREVIOUSLY UNTREATED PATIENTS; COST-MINIMIZATION ANALYSIS; HIGH-RESPONDING INHIBITORS;
D O I
10.1111/ejh.13108
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Hemophilia is associated with a high financial burden on individuals, healthcare systems, and society. The development of inhibitors significantly increases the socioeconomic burden of the diseases. This study aimed to review and describe the burden of hemophilia with inhibitors, providing a reference scenario to assess the impact of new products in the real word. Two systematic literature reviews were performed to collect data on (i) health economics and (ii) health-related quality of life evidences in hemophilic patients with inhibitors. The costs associated with patients with hemophilia and inhibitors are more than 3 times greater than the costs incurred in those without inhibitors, with an annual cost per patient that can be higher than (sic) 1000000. The costs of bypassing agents account for the large majority of the total healthcare direct costs for hemophilia treatment. The quality of life is more compromised in patients with hemophilia and inhibitors compared to those without inhibitors, in particular the physical domains, whereas mental domains were comparable to that of the general population. The development of an inhibitor has a high impact on costs and quality of life. New treatments have the potential to change positively the management and socioeconomic burden of hemophilia with inhibitors.
引用
收藏
页码:435 / 456
页数:22
相关论文
共 104 条
[11]   Cost minimization analysis to compare activated prothrombin complex concentrate (APCC) and recombinant factor VIIa for haemophilia patients with inhibitors undergoing major orthopaedic surgeries [J].
Bonnet, P. O. ;
Yoon, B. S. ;
Wong, W. -Y. ;
Boswell, K. ;
Ewenstein, B. M. .
HAEMOPHILIA, 2009, 15 (05) :1083-1089
[12]   Health-related quality of life and productivity impact in haemophilia patients with inhibitors [J].
Brown, T. M. ;
Lee, W. C. ;
Joshi, A. V. ;
Pashos, C. L. .
HAEMOPHILIA, 2009, 15 (04) :911-917
[13]   The Diagnosis and Management of Congenital Hemophilia [J].
Carcao, Manuel D. .
SEMINARS IN THROMBOSIS AND HEMOSTASIS, 2012, 38 (07) :727-734
[14]   Cost and outcome: Comparisons of two alternative bypassing agents for persons with haemophilia A complicated by an inhibitor [J].
Carlsson, Katarina Steen ;
Astermark, Jan ;
Donfield, Sharyne ;
Berntorp, Erik .
THROMBOSIS AND HAEMOSTASIS, 2008, 99 (06) :1060-1067
[15]   Haemophilia Experiences, Results and Opportunities (HERO) Study: Influence of haemophilia on interpersonal relationships as reported by adults with haemophilia and parents of children with haemophilia [J].
Cassis, F. R. M. Y. ;
Buzzi, A. ;
Forsyth, A. ;
Gregory, M. ;
Nugent, D. ;
Garrido, C. ;
Pilgaard, T. ;
Cooper, D. L. ;
Iorio, A. .
HAEMOPHILIA, 2014, 20 (04) :e287-e295
[16]   Inhibitors in haemophilia B: the Italian experience [J].
Castaman, G. ;
Bonetti, E. ;
Messina, M. ;
Morfini, M. ;
Rocino, A. ;
Scaraggi, F. A. ;
Tagariello, G. .
HAEMOPHILIA, 2013, 19 (05) :686-690
[17]   The role of recombinant activated factor VII in the haematological management of elective orthopaedic surgery in haemophilia A patients with inhibitors [J].
Castaman, Giancarlo .
BLOOD TRANSFUSION, 2017, 15 (05) :478-486
[18]  
Chang H, 1999, HAEMOPHILIA, V5, P247
[19]   Economic Burden of Illness among Persons with Hemophilia B from HUGS Vb: Examining the Association of Severity and Treatment Regimens with Costs and Annual Bleed Rates [J].
Chen, Christina X. ;
Baker, Judith R. ;
Nichol, Michael B. .
VALUE IN HEALTH, 2017, 20 (08) :1074-1082
[20]   Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial [J].
Chowdary, P. ;
Lethagen, S. ;
Friedrich, U. ;
Brand, B. ;
Hay, C. ;
Karim, F. Abdul ;
Klamroth, R. ;
Knoebl, P. ;
Laffan, M. ;
Mahlangu, J. ;
Miesbach, W. ;
Nielsen, J. Dalsgaard ;
Martin-Salces, M. ;
Angchaisuksiri, P. .
JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2015, 13 (05) :743-754