Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group (original) (raw)
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The socio-economic burden of patients affected by Hemophilia with inhibitors
European Journal of Haematology, 2018
The main clinical feature in patients affected by severe HA or HB, that is those with residual FVIII/FIX activity <1 IU/dL, 1 is recurrent spontaneous and post-traumatic bleeding, mainly occurring into joints and muscles, that seriously interfere with patients' everyday lives. 2,3 The reported hemophilia prevalence differs considerably among countries. 4 In Italy, according to the 2015 update of National Registry of Congenital Bleeding Disorders (NRCC) the prevalence is 13.5 per 100 000 males for HA (IC 95%: 13.1-14.0) and 2.9 per 100 000 males for HB (IC 95%: 2.7-3.1). 5 Further, the number of patients affected by HA recorded in the Italian NRCC was about 3.5 thousand in 2011 and over 4 thousand in 2015, this trend is due to an increase in the number of patients who are registered. Similarly, the number of patients with HB was 728 in 2011 and 859 in 2015. 6 Treatment of hemophilia is based on infusion of FVIII or FIX concentrates, given episodically (when bleeding occurs) or, prophylactically, to prevent bleeding and its deleterious effects into joints and muscles. 7 The current major complication of hemophilia treatment
Journal of Medical Economics, 2005
The effectiveness data were derived from a retrospective analysis, expert opinion and published studies. Link between effectiveness and cost data The cost analysis was carried out retrospectively on the same sample as that used for the effectiveness analysis. Study sample The study sample comprised 105 bleeding episodes in 24 patients. Bleedings were treated with rFVIIa in 28 events, with PCC in 25, with aPCC in 9, and with high-dose Factor VIII in 43. Sixteen of the 24 patients were adults aged over 17 years. The mean weight was 49 kg. No further information was given.
Economic Burden of High-Responding Inhibitors in Patients with Hemophilia A in Taiwan
Yonsei Medical Journal, 2013
Purpose: Hemophilia A (HA) is the most common X-linked inherited bleeding disorder. In some patients with HA, particularly those with severe HA, replacement therapy results in the production of high-responding clotting factor VIII inhibitors. The economic burden of this complication is the highest reported for a chronic disease. Our aim was to investigate the direct medical expenditure burden of high-responding inhibitors in patients with HA. Materials and Methods: A retrospective study was conducted using the National Health Insurance Research Database, utilizing data covering the period of 2004-2007. Results: In total, 638 males with HA¸ including 37 patients with high-responding inhibitors were evaluated. Over 99% of the annual median medical expenditure was attributable to the cost of clotting factor concentrates (CFCs) in patients with high-responding inhibitors. The annual median expenditure related to CFCs of the total medical care and outpatient care were US$170611 and US$141982, respectively, and were 4.6-and 4.3-fold higher in these patients during the study period, respectively. In patients with high-responding inhibitors, the median hospitalization expenditure and daily hospitalization cost with or without surgical procedures were 3.0-and 2.4-fold higher, respectively, and 4.3 and 5.6-fold higher, respectively. Conclusion: Our data reveal higher medical expenditures burden for patients with HA and high-responding inhibitors in Taiwan. Future research is encouraged to evaluate the impact of this burden on patient quality of life.
The development of neutralizing antibodies against factor VIII and IX complicates the clinical management of patients with hemophilia, and adds significant costs to their treatment. For this reason, several formal economic analysis have been performed comparing treatment of mild and moderate bleeding episodes in these patients in an effort to determine whether one of the two available bypassing agents would be cost-effective compared with the other. Here we review and discuss the available evidence about the economic aspects of the alternatives for on-demand treatment of mild and moderate bleeding episodes for hemophilia patients with inhibitors.
Health economics of treating haemophilia A with inhibitors
Haemophilia, 2005
Haemophilia is a rare, inherited blood disorder in which blood clotting is impaired such that patients suffer from excessive internal and external bleeding. At present there is no cure for haemophilia A and patients require expensive, lifelong treatment involving clotting factor replacement therapy. Treatment costs are perceived to be higher for patients who have developed inhibitory antibodies to factor VIII, the standard therapy for haemophilia A. However, initial cost analyses suggest that clotting factor therapy with alternative haemostatic agents, such as recombinant activated factor VII or activated prothrombin complex concentrate, is no more expensive for the majority of haemophilia A patients with inhibitors than for those without inhibitors. With the availability of effective alternative haemostatic agents, orthopaedic surgery for haemophilia A patients with inhibitors is now a clinical option, and initial cost analyses suggest this may be a cost-effective treatment strategy for patients with inhibitors whose quality of life (QoL) is severely impaired by joint arthropathy. In an era of finite healthcare resourcing it is important to determine whether new treatments justify higher unit costs compared with standard therapies and whether such higher costs are justified from an individual perspective in terms of improved QoL, and from a societal perspective in terms of improved productivity and reduced overall healthcare costs. This paper examines current data on the health economics of treating haemophilia A patients with inhibitors, focusing on the overall costs of clotting factor replacement therapy and the cost consequences of joint replacement.
Pharmacoeconomic Analysis of Hemophilia Care in Romania
2020
Hemophilia, a congenital X linked disease, has the serious burden of bleeding, requiring life-long replacement with coagulation factors (CF). In the present day, there is a continuously improving treatment for this condition. Objective: Our observational, cross-sectional study aims at finding out whether a prophylactic replacement with CF is affordable from the point of view of its cost-effectiveness in our country. Material and methods: A cohort of 122 persons with Hemophilia were included in this patient-reported outcome survey, and they answered a questionnaire consisting of 56 items, focused on 4 domains—socio-demographic, medical, quality of health/life and cost/cost-effectiveness. Results and discussion: The markers for quality of health/life were correlated with the direct and indirect costs of care, comparing subgroup 1 of patients with prophylactic vs. subgroup 2 with on-demand replacement. Based on the incremental quality adjusted life years and the incremental costs, we c...
Cost of care of haemophilia with inhibitors
Haemophilia, 2010
Summary. In Western countries, the treatment of patients with inhibitors is presently the most challenging and serious issue in haemophilia management, direct costs of clotting factor concentrates accounting for >98% of the highest economic burden absorbed for the healthcare of patients in this setting. Being designed to address questions of resource allocation and effectiveness, decision models are the golden standard to reliably assess the overall economic implications of haemophilia with inhibitors in terms of mortality, bleeding-related morbidity, and severity of arthropathy. However, presently, most data analyses stem from retrospective short-term evaluations, that only allow for the analysis of direct health costs. In the setting of chronic diseases, the cost-utility analysis, that takes into account the beneficial effects of a given treatment/healthcare intervention in terms of health-related quality of life, is likely to be the most appropriate approach. To calculate net benefits, the quality adjusted life year, that significantly reflects such health gain, has to be compared with specific economic impacts. Differences in data sources, in medical practice and/or in healthcare systems and costs, imply that most current pharmacoeconomic analyses are confined to a narrow healthcare payer perspective. Long-term/lifetime prospective or observational studies, devoted to a careful definition of when to start a treatment; of regimens (dose and type of product) to employ, and of inhibitor population (children/adults, low-responding/high responding inhibitors) to study, are thus urgently needed to allow for newer insights, based on reliable data sources into resource allocation, effectiveness and cost-utility analysis in the treatment of haemophiliacs with inhibitors.
Iranian Journal of Pharmaceutical Sciences, 2016
Bypassing agents are the most commonly used medicines for the treatment of hemophilia patients with inhibitors. The aim of this study is to identify the cost components of management of bleeding vents in hemophilia patients with inhibitors in Iran. This study is a cross-sectional study using a bottom-up approach to determine the cost components of treatment of hemophilia patients with inhibitors via ascertaining of all direct medical and non-medical costs.The evaluating of cost components of 20 patients with 40 episodes of bleeding showed that the price of medicines used is responsible for more than 96% and 97% of costs in treatment of hemophilia patients using FEIBA® and AryoSeven, respectively. The results of this study showed that cost of treating one bleeding episode in hemophilia A patients with high antibody titer using FEIBA is 376 USD compared to 857.3 USD for AryoSeven®.Despite the small number of hemophilia patients with inhibitor in Iran, due to high cost of treating thes...