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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ABSTRACT: Recombinant DNA technology and protein engineering are creating hope that we can address ongoing challenges in hemophilia care such as reducing the costs of therapy, increasing the availability to the developing world, and improving the functional properties of these proteins. Technological advances to improve the half-life of recombinant clotting factors have brought long-acting clotting factors for hemophilia replacement therapy closer to reality. Preclinical and clinical trial results are reviewed as well as the potential benefits and risks of these novel therapies.American Journal of Hematology 05/2012; 87 Suppl 1(S1):S33-9. DOI:10.1002/ajh.23146 · 3.48 Impact Factor
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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.01/2015; 2015:1-9. DOI:10.1155/2015/596164
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ABSTRACT: Objective Evaluate adherence to clotting factor treatment and associated outcomes for patients with hemophilia using an integrated delivery database. Methods This was a retrospective, observational study tracking patients between 2006 and 2011. Patients with diagnosis codes for hemophilia were identified. Bleeding and complication rates were annualized over the study period. Medication adherence was assessed using prescription claims for clotting factors by examining sequential time periods of 180 days for each patient's continuous enrollment. Adherence within the time period was calculated using the 'days supply' field divided by 180 days. Under the assumption that severe patients should be treated prophylactically, patients were considered adherent within the time period if the ratio of 'days supply' to observed days was 60% or greater. Results A total of 207 patients (74.9 and 25.1% hemophilia A and B, respectively) met the inclusion/exclusion criteria. There were 101 (48.8%) mild, 32 (15.5%) moderate, and 74 (35.7%) severe patients with hemophilia. The percentage of time periods where adherence to clotting factors was 60% or greater was 14% (SD = 28%) for mild disease, 21% (SD = 32%) for moderate disease, and 51% (SD = 36%) for severe disease. Among patients with severe disease, 27 (36.5%) were adherent ≤30% of time periods, 22 (29.7%) adherent 31-70% of the time periods, and 25 (33.8%) were adherent ≥71% of time periods. Joint bleeding episodes and hospitalizations were uncommon events among the three groups. Conclusions Among patients with severe disease, the majority (66.2%) were adherent <70% of the time.Hematology (Amsterdam, Netherlands) 07/2014; 20(3). DOI:10.1179/1607845414Y.0000000176 · 1.19 Impact Factor