Effects of secondary prophylaxis started in adolescent and adult haemophiliacs
ABSTRACT 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    . In addition, indirect costs, usually expressed in terms of considerable loss of productivity for the society, should be taken into account  . 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. "
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.01/2015; 2015:1-9. DOI:10.1155/2015/596164
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- "Pertaining to the prevention of haemophilic arthropathy , while a number of observational studies and a randomised study carried out in children with severe haemophilia have esta - blished the superiority of primary prophylaxis over on - demand treatment in reducing the risk of arthropathy ( Aledort et al , 1994 ; Liesner et al , 1996 ; Manco - Johnson et al , 2007 ) , only a few studies have evaluated the role of secondary prophylaxis in adults with severe haemophilia ( Coppola et al , 2009 ) . A retrospective survey , carried out in 84 adolescent / adult Italian haemophiliacs switched from on - demand to prophylactic treatment ( Tagliaferri et al , 2008b ) , showed that secondary prophylaxis significantly reduced the number of bleeding episodes in target joints , improving the patients ' quality of life . In spite of these findings , little is known regarding the management of arthropathy in elderly patients and further studies , dealing with the optimal regimens of replacement therapy and the development of preventive physical therapy programmes , are warranted . "
ABSTRACT: The life expectancy and quality of life of persons with haemophilia has dramatically increased since the 1970s, with the exception of the increased rate of deaths observed during the 1980s and the 1990s due to blood-borne viral infections. Improvements of factor replacement therapy, treatment of infectious diseases and comprehensive health care provided by specialised haemophilia centres are the main determinants of the increasing age of the haemophilia population. As a consequence, a growing number of these patients develop age-related co-morbidities, such as cardiovascular disease and cancer. The care of these previously rare conditions is a new challenge for caregivers in haemophilia centres. This review focuses on co-morbidities in the ageing haemophilia patients, their impact on quality of life and their complex management.British Journal of Haematology 12/2009; 148(4):522-33. DOI:10.1111/j.1365-2141.2009.08005.x · 4.96 Impact Factor
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ABSTRACT: Progressive arthropathy of large joints of the limbs (knees, ankles, elbows), resulting from recurrent joint bleeds and subsequent long-term degenerative phenomena, is one of the main causes of morbidity and of deterioration of quality of life in adult severe hemophiliacs. While primary prophylaxis (i.e. the regular continuous long-term infusion of factor concentrates started before the age of two years and/or after no more than one joint bleed) is nowadays considered the gold standard for preserving joint function in patients with severe haemophilia, the benefits of secondary prophylaxis (i.e., all the long-term regular treatments not fulfilling the criteria of primary prophylaxis) are still controversial. In this review we present the literature data on secondary prophylaxis, focusing on adolescent and adults haemophiliacs along with clinical experience in Italy. On the whole, the more recently published studies suggest the effectiveness of early and delayed secondary prophylaxis. However, a number of questions are still unanswered, including the optimal dose, dosing interval and duration of secondary prophylaxis. Only large, prospective, long-term, possibly randomized studies will help to definitively assess the clinical impact of this strategy in adolescent and adult hemophiliacs.Blood transfusion = Trasfusione del sangue 10/2008; 6 Suppl 2:s17-20. · 1.90 Impact Factor