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
[31]  
EuroQol Group, EQ-5D-5L User Guide (Version 3.0)
[32]   Spanish consensus guidelines on prophylaxis with bypassing agents for surgery in patients with haemophilia and inhibitors [J].
Eva Mingot-Castellano, Maria ;
Teresa Alvarez-Roman, Maria ;
Fernanda Lopez-Fernandez, Maria ;
Altisent-Roca, Carmen ;
Isabel Canaro-Hirnyk, Mariana ;
Jimenez-Yuste, Victor ;
Rosa Cid-Haro, Ana ;
Perez-Garrido, Rosario ;
Sedano-Balbas, Carmen .
EUROPEAN JOURNAL OF HAEMATOLOGY, 2016, 96 (05) :461-474
[33]   Inhibitor development in haemophilia according to concentrate Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project [J].
Fischer, Kathelijn ;
Lassila, Riita ;
Peyvandi, Flora ;
Calizzani, Gabriele ;
Gatt, Alex ;
Lambert, Thierry ;
Windyga, Jerzy ;
Iorio, Alfonso ;
Gilman, Estelle ;
Makris, Michael .
THROMBOSIS AND HAEMOSTASIS, 2015, 113 (05) :968-975
[34]   Inhibitor incidence in previously untreated patients with severe haemophilia B: a systematic literature review [J].
Franchini, Massimo ;
Santoro, Cristina ;
Coppola, Antonio .
THROMBOSIS AND HAEMOSTASIS, 2016, 116 (01) :201-203
[35]   Systematic Review of the Role of FVIII Concentrates in Inhibitor Development in Previously Untreated Patients with Severe Hemophilia A: A 2013 Update [J].
Franchini, Massimo ;
Coppola, Antonio ;
Rocino, Angiola ;
Santagostino, Elena ;
Tagliaferri, Annarita ;
Zanon, Ezio ;
Morfini, Massimo .
SEMINARS IN THROMBOSIS AND HEMOSTASIS, 2013, 39 (07) :752-766
[36]   Italian Registry of Congenital Bleeding Disorders [J].
Giampaolo, Adele ;
Abbonizio, Francesca ;
Arcieri, Romano ;
Hassan, Hamisa Jane .
JOURNAL OF CLINICAL MEDICINE, 2017, 6 (03)
[37]   Consensus protocol for the use of recombinant activated factor VII [eptacog alfa (activated); NovoSeven®] in elective orthopaedic surgery in haemophilic patients with inhibitors [J].
Giangrande, P. L. F. ;
Wilde, J. T. ;
Madan, B. ;
Ludlam, C. A. ;
Tuddenham, E. G. D. ;
Goddard, N. J. ;
Dolan, G. ;
Ingerslev, J. .
HAEMOPHILIA, 2009, 15 (02) :501-508
[38]  
Golestani M, 2016, IRANIAN J PHARM SCI, V12, P85
[39]  
Golestani M, 2016, IRAN J PHARM RES, V15, P669
[40]   Key issues in inhibitor management in patients with haemophilia [J].
Gomez, Keith ;
Klamroth, Robert ;
Mahlangu, Johnny ;
Mancuso, Maria E. ;
Mingot, Maria E. ;
Ozelo, Margareth Castro .
BLOOD TRANSFUSION, 2014, 12 :S319-S329