Effect of secondary prophylaxis started in adult and haemophiliacs

Regional Reference Centre for Inherited Bleeding Disorders, University Hospital, Parma, Italy.
Haemophilia (Impact Factor: 2.6). 07/2008; 14(5):945-51. DOI: 10.1111/j.1365-2516.2008.01791.x
Source: PubMed


While primary prophylaxis is a well-established and recommended method of care delivery for children with severe haemophilia, fewer studies have documented the benefits of secondary prophylaxis started in adolescence or adulthood. To evaluate the role of secondary prophylaxis started in adolescent and adult severe haemophiliacs, a retrospective observational cohort study was conducted in 10 Italian Centres that investigated 84 haemophiliacs who had bled frequently and had thus switched from on-demand to prophylactic treatment during adolescence (n = 30) or adulthood (n = 54). The consumption of clotting factor concentrates, the orthopaedic and radiological scores, quality of life and disease-related morbidity were compared before and after starting secondary prophylaxis. Prophylaxis reduced the mean annual number of total and joint bleeds (35.8 vs. 4.2 and 32.4 vs. 3.3; P < 0.01) and of days lost from work/school (34.6 vs. 3.0, P < 0.01). A statistically significant reduction in the orthopaedic score was observed during prophylaxis in adolescents, but not in the whole cohort. Patients used more factor concentrates with corresponding higher costs on prophylaxis, but experienced a better quality of life. With respect to on-demand treatment, higher factor consumption and cost of secondary prophylaxis were balanced by marked clinical benefits and greater well-being in this cohort of adolescent/adult haemophiliacs.

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    • "In particular, as confirmed by the available scientific literature, haemophilia A is one of the most expensive diseases because of the required lifetime treatment and the management of the related adverse effects and complications, especially when patients develop factor VIII inhibitors [2] [3] [4] [5]. In addition, indirect costs, usually expressed in terms of considerable loss of productivity for the society, should be taken into account [6] [7]. High healthcare costs are due to the continuous factor VIII (FVIII) infusions therapy that people affected by haemophilia A need for the management and the prevention of bleedings and to reduce the risk of complications, such as flexion contractures, joint arthritis/arthropathy, chronic pain, muscle atrophy, compartment syndrome, neurologic impairment. "
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    ABSTRACT: Objectives: Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis. The aim of the present review was to discuss and appraise the methodological aspects and results of published economic evaluations of haemophilia A treatments in the last decade. Methods: The literature search, performed by consulting four engines, covered studies published between 2002 and 2014. Full economic evaluations published in English language were identified and included in the review. A quality assessment of the studies was also carried out based on Drummond's checklist. Results: After careful evaluations of the identified records, 5 studies were reviewed. Primary and secondary prophylaxis resulted cost-effective compared to on-demand therapy: the ICER of primary prophylaxis ranged from € 40.236 to € 59.315/QALY gained, while the ICER of secondary prophylaxis was € 40.229/QALY gained. Furthermore, 60% were high quality and 40% were medium quality studies. Conclusions: The review underlines the cost-effectiveness of prophylaxis versus on-demand treatment and the different methodological approaches applied. Further economic evaluations are required with models that reflect the clinical reality and consumption of resources in each country.
    Full-text · Article · Jan 2015
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    • "Quality of life (QoL) assessment tools are being used with increasing regularity in both clinical trials and pharmacoeconomic modeling studies to unravel the potential long-term benefits of expensive procedures, such as prophylaxis, ITT, and elective orthopedic surgery. The high cost of prophylactic therapy, for example, is weighed against improvements in QoL, days gained at work and school, reduced hospitalizations, and reduced need for surgery.46–48 The European Haemophilia Economic Study Group compared the outcomes of 1033 hemophilic patients from 16 European HTCs using prophylaxis with episodic treatment.49 "
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    ABSTRACT: Early treatment of bleeds in hemophilia patients, both with and without inhibitors, has been shown to be of immense benefit in the overall clinical outcome. Despite the advantages of treating the bleeding episodes early, significant barriers and limitations remain. The aim of this review is to highlight the various barriers and perceived limitations to early therapy of bleeding episodes, especially in patients who have developed inhibitors to factor VIII. The peer-reviewed literature was searched for articles on hemophilia patients, with and without inhibitors, and early treatment, to identify the barriers to early treatment and potential impact on patient outcomes. The most important barrier is the educational barrier, which involves lack of awareness among patients regarding the signs of a bleed, as well as importance of early therapy. It is also common for parents or caregivers of school-age children to exhibit inconvenience and scheduling barriers. Distance to the treatment center can also play a role here. Some patients experience financial barriers related to cost of clotting factor products, insurance coverage, or insurance caps and out-of-pocket costs. Rarely, there can also be problems related to venous access or home infusion. Lastly, multiple psychosocial barriers can prevent adherence to treatment regimens. Identification and addressing these individual barriers will result in improved compliance rates, prevent joint damage, be more cost-effective, and lead to better overall health of these patients.
    Preview · Article · May 2013 · Hematology Research and Reviews
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    • "Tagliaferri et al assessed the effects of secondary prophylaxis started in adolescent and adult hemophiliacs. A 90% reduction in the mean annual number of joint bleeds in patients on prophylaxis accompanied an equal decrease in days lost from work or school.57 Compared to the patients on episodic factor therapy, individuals on prophylaxis had an increased consumption of factor concentrates with corresponding higher costs balanced by marked clinical benefits with improved quality of life. "
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    ABSTRACT: Individuals with severe factor VIII deficiency experience recurrent hemorrhages and develop progressive joint damage. Large retrospective, nonrandomized studies of patient cohorts followed over decades show that factor prophylaxis initiated at an early age before the onset of recurrent bleeding reduces the incidence of hemophilic arthropathy. Two recent prospective, multicenter, randomized trials conducted in Europe (the ESPRIT study) and the USA (the Joint Outcome Study) confirm the efficacy of prophylaxis in the prevention of hemarthroses and arthropathy. Regular prophylaxis initiated in early childhood enhances the quality of life for patients with severe hemophilia and reduces the risk of inhibitor development. The substantial costs of such preventative therapy may be offset by the reduced expenditures that the care of degenerative joint disease in adult hemophilia patients would otherwise require.
    Full-text · Article · Mar 2010 · Vascular Health and Risk Management
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