Cost-utility analysis of factor VIII diet therapies prepared using blood plasma vs. recombinant technique for patients with hemophilia A.

BACKGROUND: Hemophilia is known as one of the most common coagulation disorders whose treatment costs are particularly high in developing countries, and about 90% of them are related to factor VIII (FVIII) and direct medical costs (DMCs). Thus, the present study aimed to analyze cost-utility of two FVIII diet therapies prepared using blood plasma and recombinant technique. METHODS: This study was an economic evaluation fulfilled through a cost-utility approach. To this end, a total number of 120 patients were randomly selected using Krejcie & Morgan's Table and then received blood plasma and recombinant FVIII. The decision tree structure was also utilized to estimate economic and clinical outcomes. Moreover, costs were reviewed from societal perspective. Quality-adjusted life year (QALY) was subsequently determined as the measure of effectiveness (MOE). Besides, one-way (univariate) sensitivity analysis was performed to quantify uncertainty effects of the study parameters. The information was ultimately analyzed using the TreeAge Pro 2011 and the Microsoft Office Excel 2010 software. RESULTS: The results revealed that the recombinant diet therapy had higher costs and effectiveness compared with blood-plasma-derived FVIII, so that the mean costs of these two diet therapies were equal to 37,624 and 20,349 purchasing power parity (PPP) $ with utility scores of 0.78 and 0.62; respectively. Since the incremental cost-effectiveness ratio (ICER) for the recombinant medications was over three times of the threshold level, it was considered as overwhelming because of its high cost in spite of its better effectiveness. Moreover, the results of one-way (univariate) sensitivity analysis demonstrated the highest sensitivity to the utility in patients who had been injected with blood-plasma-derived FVIII and had been successfully treated. CONCLUSION: The study results revealed that FVIII prepared using blood plasma for hemophilia A patients had higher cost-effectiveness compared with that made using recombinant technique. Graphical abstract.

Factors affecting the quality, safety and marketing approval of clotting factor concentrates for haemophilia.

Selecting therapeutic products for the treatment of haemophilia follows the process of obtaining market approval of products submitted to the scrutiny of a regulatory agency. In well-resourced countries, key decisions on whether a product is sufficiently safe and of high quality are made by highly expert and well-resourced agencies, such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In countries lacking such agencies, well-informed decisions can still be made through an appreciation of the key issues affecting the quality, safety and efficacy of haemophilia products. A number of well-established principles may then be applied in order to make a choice. In this review, reflecting principles outlined by the World Federation of Hemophilia, we outline the key features in determining the acceptability of therapeutic products for haemophilia in order to ensure an optimal choice in all the environments providing haemophilia care.

[Food consumption and dietary behaviour in haemophilia patients before and after a nutrition consultation]. / Lebensmittelkonsum und Ernährungsverhalten bei Patienten mit Hämophilie vor und nach Ernährungsberatung.

UNLABELLED: In over two-thirds of deaths, nutrition is a determining factor. Nutritional condition and dietary recommandations are unspecified in the haemophilia treatment. Aim of this study was to examine the food consumption and dietary behaviour in affected patients before and after a nutrition consultation. PATIENTS, METHODS: Data were assessed via questionnaires and compared between 38 patients with haemophilia (PwH) and 20 controls without haemophilia. Furthermore, in a randomised controlled trial 11 patients with haemophilia (PwH-I) took part in an adapted nutrition consultation and were supervised over six months as opposed to 12 patients with haemophilia (PwH-O) without intervention. PwH were compared to controls more pleased with their weight (53 vs. 40%), used more nutrition consultations in the past (53 vs. 15%) and consumed more dairy products (40 vs. 15%) and fruits (45 vs. 30%). RESULTS: After nutrition consultation PwH-I were better informed about their own blood values than PwH-O. The nutrition rated high in both groups, but PwH-I were more mindful of the feeling of satiety (9 vs. 36%) compared to PwH-O (33 vs. 17%). Moreover, PwH-I ate less under stress and/or out of boredom, showed a higher satisfaction regarding their weight and increased the liquid intake (55 vs. 73%), which remained unchanged in PwH-O with 42%. Compared to PwH-O, PwH-I ate more roughage, low-fat food, fish and fruits, therefore consuming less coffee/tea. CONCLUSION: Food consumption and dietary behaviour are similar between patients with haemophilia and controls without haemophilia. A nutrition consultation affected the food consumption and dietary behaviour in patients with haemophilia positively and can consequently contribute to preservation of health and prevention of nutritional diseases.

[The alimentary status sisk of the hevophilia with the lesion of jointts].

The diagnostic findings sick of a hemophilia with recurring hemarthrosis are presented. At studying of componential structure of a body by a method of bioimpedansometriya considerable fluctuations of an index of weight of a body, a poor development of a muscular fabric, increase in percentage of a fatty fabric that is connected with low, physical activity and increase in the contribution of fat in power value of a food ration have been found out in patients. Results of research of an actual food by a frequency method with a quantitative estimation at sick of hemophilia and healthy students testify to probable risk of insufficient consumption of vitamins B1, B2 and calcium, magnesium that demands individual correction.

How I treat age-related morbidities in elderly persons with hemophilia.

In persons with hemophilia, life expectancy is now approaching that of the general male population, at least in countries that can afford regular replacement therapy with coagulation factor concentrates. The new challenges for comprehensive treatment centers are thus to provide optimal health care for this aging population of patients, who often present not only with the comorbidities typically associated with hemophilia (arthropathy, chronic pain, blood-borne infections), but also with common age-related illnesses such as cardiovascular disease and cancer. There are no evidence-based guidelines for the management of these conditions, which often require drugs that interfere with hemostasis, enhance the bleeding tendency, and warrant more intensive replacement therapy. At the moment, elderly patients with hemophilia affected by other diseases should be managed like their age-group peers without hemophilia, provided replacement therapy is tailored to the heightened risk of bleeding associated with the need for invasive procedures and drugs that further compromise the deranged hemostasis. More detailed advice is provided on the schedules of replacement therapy needed to tackle cardiovascular diseases, such as acute coronary syndromes and nonvalvular atrial fibrillation, because these conditions will become more and more frequent challenges for the comprehensive treatment centers.

Outpatient treatment of hemophiliacs for dental extractions.

The outpatient treatment program for dental extraction in persons with the various forms of hemophilia has been reported. This program was initiated at the Hemophilia Rehabilitation Center at Orthopaedic Hospital in Los Angeles in 1966. During the nine years of this program, 260 patients have had 642 teeth extracted. eighty percent of these patients were outpatients and 64% of the extractions were performed on an outpatient basis. This outpatient treatment has been accompanied by an absence of a significant number of complications as evidenced by the postextraction hospitalization of only five patients during the nine-year period. The surgical technique uses a local anesthetic containing a vasoconstrictor or an ultralight intravenous general anesthetic in addition to the local anesthetic for the apprehensive or acutely infected patient. EACA is used as an antifibrinolytic agent postsurgically. Patient education and cooperation, diet control, and daily contact are important factors for the success of an outpatient oral surgery program for hemophilic patients.