Haemophilia : the official journal of the World Federation of Hemophilia
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Creutzfeldt-Jakob disease (CJD) was first described as a clinical entity in the 1920s, first transmitted experimentally in 1968, and first transmitted iatrogenically in 1972 (corneal transplant). Numerous experimental studies in rodents, sheep and primates have since revealed very low levels of infectivity in blood (about 1/100 000th the level in brain tissue) that can appear as early as half-way through the incubation period, with 5-10 fold higher concentrations in leucocytes than plasma. ⋯ Plasma and plasma proteins have not been implicated in any transmissions, and no instance of transmission from the blood of individuals incubating other forms of CJD has been recognized. Strategies to prevent iatrogenic transmissions include low-risk sourcing, leucodepletion, and a variety of infectivity-reducing plasma processing steps; screening tests to detect infection in preclinical donors are under development.
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The first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil is currently activated prothrombin complex concentrate (aPCC), with recombinant activated factor VII (rFVIIa) used as second-line therapy or as a last resort. The aim of this study was to determine the cost and effectiveness of these treatments from the perspective of the Brazilian National Health Service. A decision analysis model was constructed to assess total direct medical costs (including drug costs, costs of outpatient or inpatient care, ambulance transportation and cost of concomitant medications) of first-line treatment with aPCC or rFVIIa. ⋯ Mean total direct medical costs (from initiation to cessation of bleed) were estimated to be US$13 500 (aPCC) and US$7590 (rFVIIa). Extensive sensitivity analyses confirmed the cost-effectiveness of rFVIIa. Compared with aPCC, rFVIIa was more effective and less expensive when used as first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil. rFVIIa should be considered a first-line treatment for the management of these patients.
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Strategies for the management of perioperative bleeding in patients with haemophilia and inhibitors have evolved rapidly as a result of the development of the bypassing agents Factor Eight Inhibitor Bypassing Activity, Anti-inhibitor Coagulant Complex (FEIBA) and activated recombinant factor VII (rFVIIa). However, there are currently no established guidelines for perioperative use of bypassing agents, and few controlled clinical studies have been carried out. Thus, case reports, such as those presented here, provide useful anecdotal evidence to guide the treatment of inhibitor patients. ⋯ Haemostatic control was achieved in all cases, and treatment regimens were generally well tolerated. One thrombotic adverse event was reported: evidence of disseminated intravascular coagulation (DIC) was found after rFVIIa use in one case, although the direct cause of DIC was unknown. The experiences in this case review demonstrate that both major and minor surgical procedures can be safely performed in patients with haemophilia and high-titre inhibitors under the cover of bypassing agents, with a high expectation of success.